654 results on '"Barohn, Richard"'
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2. Conducting a bayesian multi-armed trial with response adaptive randomization for comparative effectiveness of medications for CSPN
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Brown, Alexandra R., Gajewski, Byron J., Mudaranthakam, Dinesh Pal, Pasnoor, Mamatha, Dimachkie, Mazen M., Jawdat, Omar, Herbelin, Laura, Mayo, Matthew S., and Barohn, Richard J.
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- 2023
- Full Text
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3. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial
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Dimachkie, Mazen, Statland, Jeffrey, Pasnoor, Mamatha, Jawdat, Omar, Heim, Andrew, Ciersdorff, Ali, Sasidharan, Sandhya, Currence, Melissa, Levine, Todd, Otutoa, Rebecca, Cooper, Angelina, Mozaffar, Tahseen, Habib, Ali, Cauchi, Jonathan, Ung, Shannon, Mathew, Veena, Hernandez, Isela, Gibson, Summer, Bromberg, Mark, Mahoney, Kyle, Neate, Crystal, Janecki, Teresa, Papadakis, Mike, Freimer, Miriam, Kaschalk, MacKenzie, Heintzman, Sarah, Wicklund, Matthew, Baines, Brenna, Vareldzis, Alexa, Hyslop, Emily, Blume, Brianna, Ciafaloni, Emma, Luebbe, Elizabeth, Eichinger, Katy, Martens, William, Gregory, Stephanie, Janciuras, Joanne, Amato, Anthony, Doughty, Christopher, Roe, Kristen, Flynn, Patricia, Russo, Emily, Lloyd, Thomas, Albayda, Jemima, Tiniakou, Eleni, Thomas, Simone, Jones, Sarah, Solorzano, Guillermo, Elliott, Matthew, Burns, Ted, Crowell, Allison, Eggleston, Deborah, Wagoner, Mary, Shaibani, Aziz, Oates, Chantae, Machado, Pedro, Hanna, Michael, Greensmith, Linda, Ahmed, Mhoriam, Vivekanandam, Vinojini, Appleby, Matthew, Ransley, George, Eshun, Edwin Eshun, Skorupinska, Iwona, Germain, Louise, Laxa, Ana Marie, Pontes, Joana Roca, Bellin, Anna, Anifowoshe, Dolapo, Machado, Pedro M, McDermott, Michael P, Blaettler, Thomas, Sundgreen, Claus, Amato, Anthony A, Gibson, Summer B, Jones, Sarah M, Levine, Todd D, Lloyd, Thomas E, Shaibani, Aziz I, Rosholm, Anders, Carstensen, Tim Dehli, Bonefeld, Karen, Jørgensen, Anders Nørkær, Phonekeo, Karina, Heim, Andrew J, Herbelin, Laura, Barohn, Richard J, Hanna, Michael G, and Dimachkie, Mazen M
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- 2023
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4. Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial
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Amato, Anthony A., Benveniste, Olivier, Biliciler, Suur, Chinoy, Hector, Dimachkie, Mazen M., Edmundson, Christyn, Freimer, Miriam, Geraci, Anthony, Hussain, Yessar, Machado, Pedro, Mammen, Andrew L., Mozaffar, Tahseen, Soltanzadeh, Payam, Suresh, Niraja, van der Kooi, Anneke, Allenbach, Yves, Appleby, Matthew, Barohn, Richard J, Champtiaux, Nicolas, Doughty, Christopher, Farias, Jerrica, Farmakidis, Constantine, Habib, Ali A., Karam, Chafic, Lilleker, James, Lorusso, Samantha, Pasnoor, Mamatha, Pinal-Fernandez, Iago, Querin, Giorgia, Raaphorst, Joost, Ransley, George, Saba, Sami, Sheikh, Kazim, Snedden, Andrew, Statland, Jeffrey, Vu, Tuan, Mammen, Andrew L, Amato, Anthony A, Dimachkie, Mazen M, Lilleker, James B, Boroojerdi, Babak, Vanderkelen, Mark, Delicha, Eumorphia Maria, Koendgen, Harold, Farzaneh-Far, Ramin, Duda, Petra W, and Sayegh, Camil
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- 2023
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5. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy
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Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Maggi, Lorenzo, Baranello, Giovanni, Flanigan, Kevin M., Kuntz, Nancy L., Manzur, Adnan Y., Darras, Basil T., Kang, Peter, Mah, Jean K., Mongini, Tiziana, Ricci, Federica, Morrison, Leslie, Krzesniak-Swinarska, Monika, von der Hagen, Maja, Finkel, Richard S., Kumar, Ashutosh, Wicklund, Matthew, McDonald, Craig M., Henricson, Erik K., Schara-Schmidt, Ulrike, Wilichowski, Ekkehard, Barohn, Richard J., Statland, Jeffrey, Kirschner, Janbernd, Vita, Giuseppe, Vita, Gian Luca, Howard, James F., Jr., Hughes, Imelda, McMillan, Hugh J., Pegoraro, Elena, Bello, Luca, Burnette, W. Bryan, Thangarajh, Mathula, Chang, Taeun, Campbell, Craig, McColl, Elaine, McDermott, Michael P., Martens, William B., Guglieri, Michela, and Griggs, Robert C.
- Published
- 2021
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6. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial
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Cutter, Gary, Aban, Inmaculada, Minisman, Greg, Feese, Michelle, Kuo, Hui-Chien, Newsom-Davis, John, Wolfe, Gil, Kaminski, Henry, Jaretzki, Alfred, Sonett, Joshua, Mazia, Claudio, Saluto, Valeria, Rosenberg, Moises, Alvarez, Valeria, Rey, Lisa, King, John, Butzkueven, Helmut, Goldblatt, John, Carey, John, Pollard, John, Reddel, Stephen, Handel, Nicholas, McCaughan, Brian, Pallot, Linda, Waddington-Cruz, Márcia, Novis, Ricardo, Boasquevisque, Carlos, Dias-Tosta, Elza, Morato-Fernandez, Rubens, Ximenes, Manoel, Werneck, Lineu, Scola, Rosana, Soltoski, Paulo, Chalk, Colin, Moore, Fraser, Mulder, David, Wadup, Lisa, Oger, Joel, Mezei, Michele, Evans, Kenneth, Jiwa, Theresa, Schaffar, Anne, White, Chris, Toth, Cory, Gelfand, Gary, Wood, Susan, Pringle, Elizabeth, Zwicker, Jocelyn, Maziak, Donna, Shamji, Farid, Sundaresan, Sudhir, Seely, Andrew, Cea, Gabriel, Verduga, Renato, Aguayo, Alberto, Jander, Sebastian, Zickler, Philipp, Klein, Michael, Marx, Alexander, Ströbel, Philipp, Weis, Cleo-Aron, Melms, Arthur, Bischof, Felix, Aebert, Hermann, Ziemer, Gerhard, Nix, Wilfred, Thümler, Björn, Wilhem-Schwenkmezger, Thomas, Mayer, Eckhard, Schalke, Berthold, Pöschel, Peter, Hieber, Gisela, Wiebe, Karsten, Antonini, Giovanni, Clemenzi, Alessandro, Ceschin, Vanessa, Rendina, Erino, Venuta, Federico, Morino, Stefania, Bucci, Elisabetta, Durelli, Luca, Tavella, Alessia, Clerico, Marinella, Contessa, Giulia, Borasio, Piero, Evoli, Amelia, Servidei, Serenella, Granone, Pierluigi, Mantegazza, Renato, Berta, Emilia, Novellino, Lorenzo, Spinelli, Luisa, Motomura, Masakatsu, Matsuo, Hidenori, Nagayasu, Takeshi, Yoshikawa, Hiroaki, Takamori, Masaharu, Oda, Makoto, Matsumoto, Isao, Furukawa, Yutaka, Noto, Daisuke, Motozaki, Yuko, Iwasa, Kazuo, Yanase, Daisuke, Garcia Ramos, Guillermo, Cacho, Bernardo, de la Garza, Lorenzo, Kostera-Pruszczyk, Anne, Lipowska, Marta, Kwiecinski, Hubert, Potulska-Chromik, Anna, Orlowski, Tadeusz, Silva, Ana, Feijo, Marta, Freitas, António, Heckmann, Jeannine, Frost, Andrew, Pan, Edward, Tucker, Lawrence, Rossouw, Johan, Drummond, Fiona, Illa, Isabel, Diaz, Jorge, Leon, Carlos, Yeh, Jiann-Horng, Chiu, Hou-Chang, Hsieh, Yei-San, Witoonpanich, Rawiphan, Tunlayadechanont, Supoch, Attanavanich, Sukasom, Verschuuren, Jan, Straathof, Chiara, Titulaer, Maarten, Versteegh, Michel, Pels, Arda, Krum, Yvonne, Buckley, Camilla, Leite, M. Isabel, Vincent, Angela, Hilton-Jones, David, Ratnatunga, Chandi, Farrugia, Maria, Petty, Richard, Overell, James, Kirk, Alan, Gibson, Andrew, McDermott, Chris, Hopkinson, David, Lecky, Bryan, Watling, David, Marshall, Dot, Saminaden, Sam, Davies, Deborah, Dougan, Charlotte, Sathasivam, Siva, Page, Richard, Sussman, Jon, Ealing, John, Krysiak, Peter, Amato, Anthony, Salajegheh, Mohammad, Jaklitsch, Michael, Roe, Kristen, Ashizawa, Tetsuo, Smith, Robert Glenn, Zwischenberg, Joseph, Stanton, Penny, Barboi, Alexandru, Jaradeh, Safwan, Tisol, William, Gasparri, Mario, Haasler, George, Yellick, Mary, Dennis, Cedric, Barohn, Richard, Pasnoor, Mamatha, Dimachkie, Mazen, McVey, April, Gronseth, Gary, Dick, Arthur, Kramer, Jeffrey, Currence, Melissa, Herbelin, Laura, Belsh, Jerry, Li, George, Langenfeld, John, Mertz, Mary Ann, Benatar, Michael, Harrison, Taylor, Force, Seth, Usher, Sharon, Beydoun, Said, Lin, Frank, DeMeester, Steve, Akhter, Salem, Malekniazi, Ali, Avenido, Gina, Crum, Brian, Milone, Margherita, Cassivi, Stephen, Fisher, Janet, Ciafaloni, Emma, Heatwole, Chad, Watson, Thomas, Hilbert, James, Smirnow, Alexis, Distad, B. Jane, Weiss, Michael, Wood, Douglas, Haug, Joanna, Ernstoff, Raina, Cao, Jingyang, Chmielewski, Gary, Welsh, Robert, Duris, Robin, Gutmann, Laurie, Pawar, Gauri, Graeber, Geoffrey Marc, Altemus, Patricia, Nance, Christopher, Gutmann, Ludwig, Jackson, Carlayne, Grogan, Patrick, Calhoon, John, Kittrell, Pamela, Myers, Deborah, Hayat, Ghazala, Naunheim, Keith, Eller, Susan, Holzemer, Eve, Katirji, Bashar, Alshekhlee, Amer, Robke, Jason, Karlinchak, Brenda, Katz, Jonathan, Miller, Robert, Roan, Ralph, Forshew, Dallas, Kissel, John, Elsheikh, Bakri, Ross, Patrick, Chelnick, Sharon, Lewis, Richard, Acsadi, Agnes, Baciewicz, Frank, Masse, Stacey, Massey, Janice, Juel, Vern, Onaitis, Mark, Lowe, James, Lipscomb, Bernadette, Mozaffar, Tahseen, Thai, Gaby, Milliken, Jeffrey, Martin, Veronica, Karayan, Ronnie, Muley, Suraj, Parry, Gareth, Shumway, Sara, Oh, Shin, Claussen, Gwen, Lu, Liang, Cerfolio, Robert, Young, Angela, Morgan, Marla, Pascuzzi, Robert, Kincaid, John, Kesler, Kenneth, Guingrich, Sandy, Michaels, Angi, Phillips, Lawrence, Burns, Ted, Jones, David, Fischer, Cindy, Pulley, Michael, Berger, Alan, D'Agostino, Harry, Smith, Lisa, Rivner, Michael, Pruitt, Jerry, Landolfo, Kevin, Hillman, Demetric, Shaibani, Aziz, Sermas, Angelo, Ruel, Ross, Ismail, Farah, Sivak, Mark, Goldstein, Martin, Camunas, Jorge, Bratton, Joan, Tandan, Rup, Panitch, Hill, Leavitt, Bruce, Jones, Marilee, Muppidi, Srikanth, Vernino, Steven, Nations, Sharon, Meyer, Dan, Gorham, Nina, Wolfe, Gil I, Kaminski, Henry J, Aban, Inmaculada B, Cea, J Gabriel, Heckmann, Jeannine M, King, John O, Beydoun, Said R, Chalk, Colin H, Barboi, Alexandru C, Amato, Anthony A, Shaibani, Aziz I, Lecky, Bryan R F, Pulley, Michael T, Rivner, Michael H, Kostera-Pruszczyk, Anna, Pascuzzi, Robert M, Jackson, Carlayne E, Verschuuren, Jan J G M, Massey, Janice M, Kissel, John T, Werneck, Lineu C, Barohn, Richard J, Silvestri, Nicholas J, Conwit, Robin, Sonett, Joshua R, Jaretzki, Alfred, III, and Cutter, Gary R
- Published
- 2019
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7. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial
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De Bleecker, Jan L., De Koning, Kathy, De Mey, Katrien, De Pue, Annelien, Mercelis, Rudolf, Wyckmans, Maren, Vinck, Caroline, Wagemaekers, Linda, Baets, Jonathan, Ng, Eduardo, Shabanpour, Jafar, Daniyal, Lubna, Mannan, Shabber, Katzberg, Hans D., Genge, Angela, Siddiqi, Zaeem, Junkerová, Jana, Horakova, Jana, Reguliova, Katerina, Tyblova, Michaela, Jurajdova, Ivana, Novakova, Iveta, Jakubikova, Michala, Pitha, Jiri, Vohanka, Stanislav, Havelkova, Katerina, Horak, Tomas, Bednarik, Josef, Horakova, Mageda, Meisel, Andreas, Remstedt, Dike, Heibutzki, Claudia, Kohler, Siegfried, Gerischer, Lea, Hoffman, Sarah, Stascheit, Frauke, Vissing, John, Zafirakos, Lizzie, Khatri, Kuldeep Kumar, Autzen, Anne, Godtfeldt Stemmerik, Mads Peter, Andersen, Henning, Attarian, Shahram, Salort-Campana, Emmanuelle, Delmont, Emilien, Grapperon, Aude-Marie, Kouton, Ludivine, Tsiskaridze, Alexander, Rózsa, Csilla, Jakab, Gedeonne Margo, Toth, Szilvia, Szabo, Gyorgyi, Bors, David, Szabo, Eniko, Campanella, Angela, Vanoli, Fiammetta, Frangiamore, Rita, Antozzi, Carlo, Bonanno, Silvia, Maggi, Lorenzo, Giossi, Riccardo, Saccà, Francesco, Marsili, Angela, Pane, Chiara, Puorro, Giorgia, Reia, Antonio, Antonini, Giovanni, Alfieri, Girolamo, Morino, Stefania, Garibaldi, Matteo, Fionda, Laura, Leonardi, Luca, Konno, Shingo, Uzawa, Akiyuki, Sakuma, Kaoru, Watanabe, Chiho, Ozawa, Yukiko, Yasuda, Manato, Onishi, Yosuke, Samukawa, Makoto, Tsuda, Tomoko, Suzuki, Yasushi, Ishida, Sayaka, Watanabe, Genya, Takahashi, Masanori, Nakamura, Hiroko, Sugano, Erina, Kubota, Tomoya, Imai, Tomihiro, Suzuki., Mari, Mori, Ayako, Yamamoto, Daisuke, Ikeda, Kazuna, Hisahara, Shin, Masuda, Masayuki, Takaki, Miki, Minemoto, Kanako, Ido, Nobuhiro, Naito, Makiko, Okubo, Yoshihiko, Sugimoto, Takamichi, Takematsu, Yuka, Kamei, Ayumi, Shimizu, Mihiro, Naito, Hiroyuki, Nomura, Eiichi, Van Heur, Marjolein, Peters, Anne-Marie, Tannemaat, Martijn, Ruiter, Annabel, Keene, Kevin, Halas, Marek, Szczudlik, Andrzej, Pinkosz, Marta, Frasinska, Monika, Zwolinska, Grazyna, Kostera-Pruszczyk, Anna, Golenia, Aleksandra, Szczudlik, Piotr, Szczechowski, Lech, Pasko, Aneta, Poverennova, Irina, Urtaeva, Lubov, Kuznetsova, Nadezhda, Romanova, Tatiana, Nadezhda, Malkova, Lapochka, Elena, Korobko, Denis, Vergunova, Ilona, Melnikova, Anna, Bulatova, Ekaterina, Antipenko, Elena, Basta, Ivana, Bozovic, Ivo, Lavrnic, Dragana, Stojanovic, Vidosava Rakocevic, Beydoun, Said, Akhter, Salma, Malekniazi, Ali, Darki, Leila, Pimentel, Norianne, Cannon, Victoria, Chopra, Manisha, Traub, Rebecca, Mozaffar, Tahseen, Hernandez, Isela, Turner, Ivonne, Habib, Ali, Goyal, Namita, Kak, Manisha, Velasquez, Erik, Lam, Lucy, Suresh, Niraja, Farias, Jerrica, Jones, Sarah, Wagoner, Mary, Eggleston, Debbie, Bertorini, Tulio, Benzel, Cindy, Henegar, Robert, Pillai, Rekha, Bharavaju-Sanka, Ratna, Paiz, Carolyn, Jackson, Carlayne, Ruzhansky, Katherine, Dimitrova, Diana, Visser, Amy, Chahin, Nizar, Levine, Todd, Lisak, Robert, Jia, Kelly, Mada, Flicia, Bernitsas, Evanthia, Pasnoor, Mamatha, Roath, Katherine, Colgan, Samantha, Currence, Melissa, Heim, Andrew, Barohn, Richard, Dimachkie, Mazen, Statland, Jeffrey, Jawdat, Omar, Jabari, Duaa, Farmakidis, Constantine, Gilchrist, James, Li, Yuebing, Caristo, Irys, Hastings, Debbie, Morren, John Anthony, Weiss, Michael, Muppidi, Srikanth, Nguyen, Tia, Welsh, Lesly, So, Yuen, Goyal, Neelam, Pulley, Michael, Bailey, Cathy, Quraishi, Zubair, Berger, Alan, Sahagian, Gregory, Camberos, Yasmin, Frishberg, Benjamin, Howard, James F, Jr, Bril, Vera, Vu, Tuan, Karam, Chafic, Peric, Stojan, Margania, Temur, Murai, Hiroyuki, Bilinska, Malgorzata, Shakarishvili, Roman, Smilowski, Marek, Guglietta, Antonio, Ulrichts, Peter, Vangeneugden, Tony, Utsugisawa, Kimiaki, Verschuuren, Jan, and Mantegazza, Renato
- Published
- 2021
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8. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
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Dimachkie, Mazen M., Barohn, Richard J., Byrne, Barry, Goker-Alpan, Ozlem, Kishnani, Priya S., Ladha, Shafeeq, Laforêt, Pascal, Mengel, Karl Eugen, Peña, Loren D.M., Sacconi, Sabrina, Straub, Volker, Trivedi, Jaya, Van Damme, Philip, van der Ploeg, Ans T., Vissing, John, Young, Peter, Haack, Kristina An, Foster, Meredith, Gilbert, Jane M., Miossec, Patrick, Vitse, Olivier, Zhou, Tianyue, and Schoser, Benedikt
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- 2022
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9. Oxaloacetate treatment preserves motor function in SOD1G93A mice and normalizes select neuroinflammation-related parameters in the spinal cord
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Tungtur, Sudheer K., Wilkins, Heather M., Rogers, Robert S., Badawi, Yomna, Sage, Jessica M., Agbas, Abdulbaki, Jawdat, Omar, Barohn, Richard J., Swerdlow, Russell H., and Nishimune, Hiroshi
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- 2021
- Full Text
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10. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study
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Mazia, Claudio Gabriel, Wilken, Miguel, Barroso, Fabio, Saba, Juliet, Rugiero, Marcelo, Bettini, Mariela, Chaves, Marcelo, Vidal, Gonzalo, Garcia, Alejandra Dalila, De Bleecker, Jan, Van den Abeele, Guy, de Koning, Kathy, De Mey, Katrien, Mercelis, Rudy, Mahieu, Délphine, Wagemaekers, Linda, Van Damme, Philip, Depreitere, Annelies, Schotte, Caroline, Smetcoren, Charlotte, Stevens, Olivier, Van Daele, Sien, Vandenbussche, Nicolas, Vanhee, Annelies, Verjans, Sarah, Vynckier, Jan, D'Hondt, Ann, Tilkin, Petra, Alves de Siqueira Carvalho, Alzira, Dias Brockhausen, Igor, Feder, David, Ambrosio, Daniel, César, Pamela, Melo, Ana Paula, Martins Ribeiro, Renata, Rocha, Rosana, Bezerra Rosa, Bruno, Veiga, Thabata, da Silva, Luiz Augusto, Santos Engel, Murilo, Gonçalves Geraldo, Jordana, Ananias Morita, Maria da Penha, Nogueira Coelho, Erica, Paiva, Gabriel, Pozo, Marina, Prando, Natalia, Martineli Torres, Debora Dada, Butinhao, Cristiani Fernanda, Duran, Gustavo, Gomes da Silva, Tamires Cristina, Otavio Maia Gonçalves, Luiz, Pazetto, Lucas Eduardo, Fialho, Tomás Augusto Suriane, Renata Cubas Volpe, Luciana, Souza Duca, Luciana, Souza Bulle Oliveira, Acary, Amaral Andrade, Ana Carolina, Annes, Marcelo, Duarte Silva, Liene, Cavalcante Lino, Valeria, Pinto, Wladimir, Assis, Natália, Carrara, Fernanda, Miranda, Carolina, Souza, Iandra, Fernandes, Patricia, Siddiqi, Zaeem, Phan, Cecile, Narayan, Jeffrey, Blackmore, Derrick, Mallon, Ashley, Roderus, Rikki, Watt, Elizabeth, Junkerova, Jana, Kurkova, Barbora, Reguliova, Katarina, Zapletalova, Olga, Pitha, Jiri, Novakova, Iveta, Tyblova, Michaela, Jurajdova, Ivana, Wolfova, Marcela, Andersen, Henning, Harbo, Thomas, Vinge, Lotte, Krogh, Susanne, Mogensen, Anita, Vissing, John, Højgaard, Joan, Witting, Nanna, Mette Ostergaard Autzen, Anne, Pedersen, Jane, Eralinna, Juha-Pekka, Laaksonen, Mikko, Oksaranta, Olli, Harrison, Tuula, Eriksson, Jaana, Rozsa, Csilla, Horvath, Melinda, Lovas, Gabor, Matolcsi, Judit, Szabo, Gyorgyi, Jakab, Gedeonne, Szabadosne, Brigitta, Antonini, Giovanni, Di Pasquale, Antonella, Garibaldi, Matteo, Morino, Stefania, Troili, Fernanda, Fionda, Laura, Filla, Allessandro, Costabile, Teresa, Marano, Enrico, Saccà, Francesco, Fasanaro, Angiola, Marsili, Angela, Puorro, Giorgia, Mantegazza, Renato, Antozzi, Carlo, Bonanno, Silvia, Camera, Giorgia, Locatelli, Alberta, Maggi, Lorenzo, Pasanisi, Maria, Campanella, Angela, Evoli, Amelia, Alboini, Paolo Emilio, D'Amato, Valentina, Iorio, Raffaele, Kanai, Tetsuya, Kawaguchi, Naoki, Mori, Masahiro, Kaneko, Yoko, Kanzaki, Akiko, Kobayashi, Eri, Masaki, Katsuhisa, Matsuse, Dai, Matsushita, Takuya, Uehara, Taira, Shimpo, Misa, Jingu, Maki, Kikutake, Keiko, Nakamura, Yumiko, Sano, Yoshiko, Nagane, Yuriko, Kamegamori, Ikuko, Tsuda, Tomoko, Fujii, Yuko, Futono, Kazumi, Ozawa, Yukiko, Mizugami, Aya, Saito, Yuka, Morikawa, Miyuki, Samukawa, Makoto, Kamakura, Sachiko, Miyawaki, Eriko, Mitazaki, Teiichiro, Motomura, Masakatsu, Mukaino, Akihiro, Yoshimura, Shunsuke, Asada, Shizuka, Yoshida, Seiko, Amamoto, Shoko, Kobashikawa, Tomomi, Koga, Megumi, Maeda, Yasuko, Takada, Kazumi, Takada, Mihoko, Tsurumaru, Masako, Yamashita, Yumi, Akiyama, Tetsuya, Narikawa, Koichi, Tano, Ohito, Tsukita, Kenichi, Kurihara, Rikako, Meguro, Fumie, Fukuda, Yusuke, Sato, Miwako, Funaka, Soichiro, Kawamura, Tomohiro, Makamori, Masayuki, Takahashi, Masanori, Taichi, Namie, Hasuike, Tomoya, Higuchi, Eriko, Kobayashi, Hisako, Osakada, Kaori, Tsuda, Emiko, Shimohama, Shun, Hayashi, Takashi, Hisahara, Shin, Kawamata, Jun, Murahara, Takashi, Saitoh, Masaki, Suzuki, Shuichiro, Yamamoto, Daisuke, Ishiyama, Yoko, Ishiyama, Naoko, Noshiro, Mayuko, Takeyama, Rumi, Uwasa, Kaori, Yasuda, Ikuko, van der Kooi, Anneke, de Visser, Marianne, Gibson, Tamar, Casasnovas, Carlos, Alberti Aguilo, Maria Antonia, Homedes-Pedret, Christian, Julia Palacios, Natalia, Diez Porras, Laura, Velez Santamaria, Valentina, Lazaro, Ana, Diez Tejedor, Exuperio, Gomez Salcedo, Pilar, Fernandez-Fournier, Mireya, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Sastre, Maria, Gamez, Josep, Sune, Pilar, Salvado, Maria, Gili, Gisela, Mazuela, Gonzalo, Illa, Isabel, Cortes Vicente, Elena, Diaz-Manera, Jordi, Querol Gutierrez, Luis Antonio, Rojas Garcia, Ricardo, Vidal, Nuria, Arribas-Ibar, Elisabet, Piehl, Fredrik, Hietala, Albert, Bjarbo, Lena, Sengun, Ihsan, Meherremova, Arzu, Ozcelik, Pinar, Balkan, Bengu, Tuga, Celal, Ugur, Muzeyyen, Erdem-Ozdamar, Sevim, Bekircan-Kurt, Can Ebru, Acar, Nazire Pinar, Yilmaz, Ezgi, Caliskan, Yagmur, Orsel, Gulsah, Efendi, Husnu, Aydinlik, Seda, Cavus, Hakan, Kutlu, Ayse, Becerikli, Gulsah, Semiz, Cansu, Tun, Ozlem, Terzi, Murat, Dogan, Baki, Onar, Musa Kazim, Sen, Sedat, Kirbas Cavdar, Tugce, Veske, Adife, Norwood, Fiona, Dimitriou, Aikaterini, Gollogly, Jakit, Mahdi-Rogers, Mohamed, Seddigh, Arshira, Sokratous, Giannis, Maier, Gal, Sohail, Faisal, Jacob, Saiju, Sadalage, Girija, Torane, Pravin, Brown, Claire, Shah, Amna, Sathasivam, Sivakumar, Arndt, Heike, Davies, Debbie, Watling, Dave, Amato, Anthony, Cochrane, Thomas, Salajegheh, Mohammed, Roe, Kristen, Amato, Katherine, Toska, Shirli, Wolfe, Gil, Silvestri, Nicholas, Patrick, Kara, Zakalik, Karen, Katz, Jonathan, Miller, Robert, Engel, Marguerite, Forshew, Dallas, Bravver, Elena, Brooks, Benjamin, Plevka, Sarah, Burdette, Maryanne, Cunningham, Scott, Sanjak, Mohammad, Kramer, Megan, Nemeth, Joanne, Schommer, Clara, Tierney, Scott, Juel, Vern, Guptill, Jeffrey, Hobson-Webb, Lisa, Massey, Janice, Beck, Kate, Carnes, Donna, Loor, John, Anderson, Amanda, Pascuzzi, Robert, Bodkin, Cynthia, Kincaid, John, Snook, Riley, Guingrich, Sandra, Micheels, Angela, Chaudhry, Vinay, Corse, Andrea, Mosmiller, Betsy, Kelley, Andrea, Ho, Doreen, Srinivasan, Jayashri, Vytopil, Michal, Jara, Jordan, Ventura, Nicholas, Scala, Stephanie, Carter, Cynthia, Donahue, Craig, Herbert, Carol, Weiner, Elaine, Alam, Sharmeen, McKinnon, Jonathan, Haar, Laura, McKinnon, Naya, Alcon, Karan, McKenna, Kaitlyn, Sattar, Nadia, Daniels, Kevin, Jeffery, Dennis, Freimer, Miriam, Hoyle, Joseph Chad, Kissel, John, Agriesti, Julie, Chelnick, Sharon, Mezache, Louisa, Pineda, Colleen, Muharrem, Filiz, Karam, Chafic, Khoury, Julie, Marburger, Tessa, Kaur, Harpreet, Dimitrova, Diana, Gilchrist, James, Agrawal, Brajesh, Elsayed, Mona, Kohlrus, Stephanie, Andoin, Angela, Darnell, Taylor, Golden, Laura, Lokaitis, Barbara, Seelback, Jenna, Muppidi, Srikanth, Goyal, Neelam, Sakamuri, Sarada, So, Yuen T., Paulose, Shirley, Pol, Sabrina, Welsh, Lesly, Bhavaraju-Sanka, Ratna, Tobon Gonzales, Alejandro, Dishman, Lorraine, Jones, Floyd, Gonzalez, Anna, Padilla, Patricia, Saklad, Amy, Silva, Marcela, Kazamel, Mohamed, Alsharabati, Mohammad, Lu, Liang, Nozaki, Kenkichi, Mumfrey-Thomas, Sandi, Woodall, Amy, Mozaffar, Tahseen, Cash, Tiyonnoh, Goyal, Namita, Roy, Gulmohor, Mathew, Veena, Maqsood, Fatima, Minton, Brian, Jones, H. James, Rosenfeld, Jeffrey, Garcia, Rebekah, Echevarria, Laura, Garcia, Sonia, Pulley, Michael, Aranke, Shachie, Berger, Alan Ross, Shah, Jaimin, Shabbir, Yasmeen, Smith, Lisa, Varghese, Mary, Gutmann, Laurie, Gutmann, Ludwig, Jerath, Nivedita, Nance, Christopher, Swenson, Andrea, Olalde, Heena, Kressin, Nicole, Sieren, Jeri, Barohn, Richard, Dimachkie, Mazen, Glenn, Melanie, McVey, April, Pasnoor, Mamatha, Statland, Jeffery, Wang, Yunxia, Liu, Tina, Emmons, Kelley, Jenci, Nicole, Locheke, Jerry, Fondaw, Alex, Johns, Kathryn, Rico, Gabrielle, Walsh, Maureen, Herbelin, Laura, Hafer-Macko, Charlene, Kwan, Justin, Zilliox, Lindsay, Callison, Karen, Young, Valerie, DiSanzo, Beth, Naunton, Kerry, Benatar, Michael, Bilsker, Martin, Sharma, Khema, Cooley, Anne, Reyes, Eliana, Michon, Sara-Claude, Sheldon, Danielle, Steele, Julie, Howard Jr, James, Chopra, Manisha, Traub, Rebecca, Vu, Tuan, Katzin, Lara, McClain, Terry, Harvey, Brittany, Hart, Adam, Huynh, Kristin, Beydoun, Said, Chilingaryan, Amaiak, Doan, Victor, Droker, Brian, Gong, Hui, Karimi, Sanaz, Lin, Frank, Pokala, Krishna, Shah, Akshay, Tran, Anh, Akhter, Salma, Malekniazi, Ali, Tandan, Rup, Hehir, Michael, Waheed, Waqar, Lucy, Shannon, Weiss, Michael, Distad, Jane, Strom, Susan, Downing, Sharon, Kim, Bryan, Nowak, Richard, Dicapua, Daniel, Keung, Benison, Kumar, Aditya, Patwa, Huned, Robeson, Kimberly, Yang, Irene, Nye, Joan, Vu, Hong, Murai, Hiroyuki, Uzawa, Akiyuki, Suzuki, Yasushi, Imai, Tomihiro, Shiraishi, Hirokazu, Suzuki, Hidekazu, Okumura, Meinoshin, O’Brien, Fanny, Wang, Jing-Jing, Fujita, Kenji P., and Utsugisawa, Kimiaki
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- 2019
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11. A Phonetic Complexity-Based Approach for Intelligibility and Articulatory Precision Testing: A Preliminary Study on Talkers with Amyotrophic Lateral Sclerosis
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Kuruvilla-Dugdale, Mili, Custer, Claire, Heidrick, Lindsey, Barohn, Richard, and Govindarajan, Raghav
- Abstract
Purpose: This study describes a phonetic complexity-based approach for speech intelligibility and articulatory precision testing using preliminary data from talkers with amyotrophic lateral sclerosis. Method: Eight talkers with amyotrophic lateral sclerosis and 8 healthy controls produced a list of 16 low and high complexity words. Sixty-four listeners judged the samples for intelligibility, and 2 trained listeners completed phoneme-level analysis to determine articulatory precision. To estimate percent intelligibility, listeners orthographically transcribed each word, and the transcriptions were scored as being either accurate or inaccurate. Percent articulatory precision was calculated based on the experienced listeners' judgments of phoneme distortions, deletions, additions, and/or substitutions for each word. Articulation errors were weighted based on the perceived impact on intelligibility to determine word-level precision. Results: Between-groups differences in word intelligibility and articulatory precision were significant at lower levels of phonetic complexity as dysarthria severity increased. Specifically, more severely impaired talkers showed significant reductions in word intelligibility and precision at both complexity levels, whereas those with milder speech impairments displayed intelligibility reductions only for more complex words. Articulatory precision was less sensitive to mild dysarthria compared to speech intelligibility for the proposed complexity-based approach. Conclusions: Considering phonetic complexity for dysarthria tests could result in more sensitive assessments for detecting and monitoring dysarthria progression.
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- 2018
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12. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial
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Hanna, Michael G, Badrising, Umesh A, Benveniste, Olivier, Lloyd, Thomas E, Needham, Merrilee, Chinoy, Hector, Aoki, Masashi, Machado, Pedro M, Liang, Christina, Reardon, Katrina A, de Visser, Marianne, Ascherman, Dana P, Barohn, Richard J, Dimachkie, Mazen M, Miller, James A L, Kissel, John T, Oskarsson, Björn, Joyce, Nanette C, Van den Bergh, Peter, Baets, Jonathan, De Bleecker, Jan L, Karam, Chafic, David, William S, Mirabella, Massimiliano, Nations, Sharon P, Jung, Hans H, Pegoraro, Elena, Maggi, Lorenzo, Rodolico, Carmelo, Filosto, Massimiliano, Shaibani, Aziz I, Sivakumar, Kumaraswamy, Goyal, Namita A, Mori-Yoshimura, Madoka, Yamashita, Satoshi, Suzuki, Naoki, Katsuno, Masahisa, Murata, Kenya, Nodera, Hiroyuki, Nishino, Ichizo, Romano, Carla D, Williams, Valerie S L, Vissing, John, Auberson, Lixin Zhang, Wu, Min, de Vera, Ana, Papanicolaou, Dimitris A, and Amato, Anthony A
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- 2019
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13. Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study
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Nowak, Richard J., Coffey, Christopher S., Goldstein, Jonathan M., Dimachkie, Mazen M., Benatar, Michael, Kissel, John T., Wolfe, Gil I., Burns, Ted M., Freimer, Miriam L., Nations, Sharon, Granit, Volkan, Smith, A. Gordon, Richman, David P., Ciafaloni, Emma, Al-Lozi, Muhammad T., Sams, Laura Ann, Quan, Dianna, Ubogu, Eroboghene, Pearson, Brenda, Sharma, Aditi, Yankey, Jon W., Uribe, Liz, Shy, Michael, Amato, Anthony A., Conwit, Robin, OʼConnor, Kevin C., Hafler, David A., Cudkowicz, Merit E., and Barohn, Richard J.
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- 2022
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14. Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis—insights from a completed randomized controlled trial with rasagiline.
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Witzel, Simon, Statland, Jeffrey M., Steinacker, Petra, Otto, Markus, Dorst, Johannes, Schuster, Joachim, Barohn, Richard J., and Ludolph, Albert C.
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AMYOTROPHIC lateral sclerosis ,RANDOMIZED controlled trials ,CYTOPLASMIC filaments ,CLUSTER randomized controlled trials ,DISEASE progression ,DISEASE duration - Abstract
Background and purpose: Rasagiline might be disease modifying in patients with amyotrophic lateral sclerosis (ALS). The aim was to evaluate the effect of rasagiline 2 mg/day on neurofilament light chain (NfL), a prognostic biomarker in ALS. Methods: In 65 patients with ALS randomized in a 3:1 ratio to rasagiline 2 mg/day (n = 48) or placebo (n = 17) in a completed randomized controlled multicentre trial, NfL levels in plasma were measured at baseline, month 6 and month 12. Longitudinal changes in NfL levels were evaluated regarding treatment and clinical parameters. Results: Baseline NfL levels did not differ between the study arms and correlated with disease progression rates both pre‐baseline (r = 0.64, p < 0.001) and during the study (r = 0.61, p < 0.001). NfL measured at months 6 and 12 did not change significantly from baseline in both arms, with a median individual NfL change of +1.4 pg/mL (interquartile range [IQR] −5.6, 14.2) across all follow‐up time points. However, a significant difference in NfL change at month 12 was observed between patients with high and low NfL baseline levels treated with rasagiline (high [n = 13], −6.9 pg/mL, IQR −20.4, 6.0; low [n = 18], +5.9 pg/mL, IQR −1.4, 19.7; p = 0.025). Additionally, generally higher longitudinal NfL variability was observed in patients with high baseline levels, whereas disease progression rates and disease duration at baseline had no impact on the longitudinal NfL course. Conclusion: Post hoc NfL measurements in completed clinical trials are helpful in interpreting NfL data from ongoing and future interventional trials and could provide hypothesis‐generating complementary insights. Further studies are warranted to ultimately differentiate NfL response to treatment from other factors. [ABSTRACT FROM AUTHOR]
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- 2024
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15. Inclusion Body Myositis: Update on Pathogenesis and Treatment
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Naddaf, Elie, Barohn, Richard J., and Dimachkie, Mazen M.
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- 2018
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16. Genome-wide Analyses Identify KIF5A as a Novel ALS Gene
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Logullo, Francesco O., Simone, Isabella, Logroscino, Giancarlo, Salvi, Fabrizio, Bartolomei, Ilaria, Borghero, Giuseppe, Murru, Maria Rita, Costantino, Emanuela, Pani, Carla, Puddu, Roberta, Caredda, Carla, Piras, Valeria, Tranquilli, Stefania, Cuccu, Stefania, Corongiu, Daniela, Melis, Maurizio, Milia, Antonio, Marrosu, Francesco, Marrosu, Maria Giovanna, Floris, Gianluca, Cannas, Antonino, Capasso, Margherita, Caponnetto, Claudia, Mancardi, Gianluigi, Origone, Paola, Mandich, Paola, Conforti, Francesca L., Cavallaro, Sebastiano, Mora, Gabriele, Marinou, Kalliopi, Sideri, Riccardo, Penco, Silvana, Mosca, Lorena, Lunetta, Christian, Pinter, Giuseppe Lauria, Corbo, Massimo, Riva, Nilo, Carrera, Paola, Volanti, Paolo, Mandrioli, Jessica, Fini, Nicola, Fasano, Antonio, Tremolizzo, Lucio, Arosio, Alessandro, Ferrarese, Carlo, Trojsi, Francesca, Tedeschi, Gioacchino, Monsurrò, Maria Rosaria, Piccirillo, Giovanni, Femiano, Cinzia, Ticca, Anna, Ortu, Enzo, La Bella, Vincenzo, Spataro, Rossella, Colletti, Tiziana, Sabatelli, Mario, Zollino, Marcella, Conte, Amelia, Luigetti, Marco, Lattante, Serena, Marangi, Giuseppe, Santarelli, Marialuisa, Petrucci, Antonio, Pugliatti, Maura, Pirisi, Angelo, Parish, Leslie D., Occhineri, Patrizia, Giannini, Fabio, Battistini, Stefania, Ricci, Claudia, Benigni, Michele, Cau, Tea B., Loi, Daniela, Calvo, Andrea, Moglia, Cristina, Brunetti, Maura, Barberis, Marco, Restagno, Gabriella, Casale, Federico, Marrali, Giuseppe, Fuda, Giuseppe, Ossola, Irene, Cammarosano, Stefania, Canosa, Antonio, Ilardi, Antonio, Manera, Umberto, Grassano, Maurizio, Tanel, Raffaella, Pisano, Fabrizio, Harms, Matthew B., Goldstein, David B., Shneider, Neil A., Goutman, Stephen, Simmons, Zachary, Miller, Timothy M., Chandran, Siddharthan, Pal, Suvankar, Manousakis, Georgios, Appel, Stanley H., Simpson, Ericka, Wang, Leo, Baloh, Robert H., Gibson, Summer, Bedlack, Richard, Lacomis, David, Sareen, Dhruv, Sherman, Alexander, Bruijn, Lucie, Penny, Michelle, Allen, Andrew S., Appel, Stanley, Bedlack, Richard S., Boone, Braden E., Brown, Robert, Carulli, John P., Chesi, Alessandra, Chung, Wendy K., Cirulli, Elizabeth T., Cooper, Gregory M., Couthouis, Julien, Day-Williams, Aaron G., Dion, Patrick A., Gitler, Aaron D., Glass, Jonathan D., Han, Yujun, Harris, Tim, Hayes, Sebastian D., Jones, Angela L., Keebler, Jonathan, Krueger, Brian J., Lasseigne, Brittany N., Levy, Shawn E., Lu, Yi-Fan, Maniatis, Tom, McKenna-Yasek, Diane, Myers, Richard M., Petrovski, Slavé, Pulst, Stefan M., Raphael, Alya R., Ravits, John M., Ren, Zhong, Rouleau, Guy A., Sapp, Peter C., Sims, Katherine B., Staropoli, John F., Waite, Lindsay L., Wang, Quanli, Wimbish, Jack R., Xin, Winnie W., Phatnani, Hemali, Kwan, Justin, Broach, James R., Arcila-Londono, Ximena, Lee, Edward B., Van Deerlin, Vivianna M., Fraenkel, Ernest, Ostrow, Lyle W., Baas, Frank, Zaitlen, Noah, Berry, James D., Malaspina, Andrea, Fratta, Pietro, Cox, Gregory A., Thompson, Leslie M., Finkbeiner, Steve, Dardiotis, Efthimios, Hornstein, Eran, MacGowan, Daniel J., Heiman-Patterson, Terry, Hammell, Molly G., Patsopoulos, Nikolaos A., Dubnau, Joshua, Nath, Avindra, Kaye, Julia, Finkbeiner, Steven, Wyman, Stacia, LeNail, Alexander, Lima, Leandro, Rothstein, Jeffrey D., Svendsen, Clive N., Van Eyk, Jenny, Maragakis, Nicholas J., Kolb, Stephen J., Cudkowicz, Merit, Baxi, Emily, Benatar, Michael, Taylor, J. Paul, Wu, Gang, Rampersaud, Evadnie, Wuu, Joanne, Rademakers, Rosa, Züchner, Stephan, Schule, Rebecca, McCauley, Jacob, Hussain, Sumaira, Cooley, Anne, Wallace, Marielle, Clayman, Christine, Barohn, Richard, Statland, Jeffrey, Ravits, John, Swenson, Andrea, Jackson, Carlayne, Trivedi, Jaya, Khan, Shaida, Katz, Jonathan, Jenkins, Liberty, Burns, Ted, Gwathmey, Kelly, Caress, James, McMillan, Corey, Elman, Lauren, Pioro, Erik, Heckmann, Jeannine, So, Yuen, Walk, David, Maiser, Samuel, Zhang, Jinghui, Silani, Vincenzo, Ticozzi, Nicola, Gellera, Cinzia, Ratti, Antonia, Taroni, Franco, Lauria, Giuseppe, Verde, Federico, Fogh, Isabella, Tiloca, Cinzia, Comi, Giacomo P., Sorarù, Gianni, Cereda, Cristina, D’Alfonso, Sandra, Corrado, Lucia, De Marchi, Fabiola, Corti, Stefania, Ceroni, Mauro, Mazzini, Letizia, Siciliano, Gabriele, Filosto, Massimiliano, Inghilleri, Maurizio, Peverelli, Silvia, Colombrita, Claudia, Poletti, Barbara, Maderna, Luca, Del Bo, Roberto, Gagliardi, Stella, Querin, Giorgia, Bertolin, Cinzia, Pensato, Viviana, Castellotti, Barbara, Camu, William, Mouzat, Kevin, Lumbroso, Serge, Corcia, Philippe, Meininger, Vincent, Besson, Gérard, Lagrange, Emmeline, Clavelou, Pierre, Guy, Nathalie, Couratier, Philippe, Vourch, Patrick, Danel, Véronique, Bernard, Emilien, Lemasson, Gwendal, Al Kheifat, Ahmad, Al-Chalabi, Ammar, Andersen, Peter, Basak, A. Nazli, Blair, Ian P., Chio, Adriano, Cooper-Knock, Jonathan, de Carvalho, Mamede, Dekker, Annelot, Drory, Vivian, Redondo, Alberto Garcia, Gotkine, Marc, Hardiman, Orla, Hide, Winston, Iacoangeli, Alfredo, Glass, Jonathan, Kenna, Kevin, Kiernan, Matthew, Kooyman, Maarten, Landers, John, McLaughlin, Russell, Middelkoop, Bas, Mill, Jonathan, Neto, Miguel Mitne, Moisse, Mattieu, Pardina, Jesus Mora, Morrison, Karen, Newhouse, Stephen, Pinto, Susana, Pulit, Sara, Robberecht, Wim, Shatunov, Aleksey, Shaw, Pamela, Shaw, Chris, Sproviero, William, Tazelaar, Gijs, van Damme, Philip, van den Berg, Leonard, van der Spek, Rick, van Eijk, Kristel, van Es, Michael, van Rheenen, Wouter, van Vugt, Joke, Veldink, Jan, Weber, Markus, Williams, Kelly L., Zatz, Mayana, Bauer, Denis C., Twine, Natalie A., Nicolas, Aude, Kenna, Kevin P., Renton, Alan E., Faghri, Faraz, Chia, Ruth, Dominov, Janice A., Kenna, Brendan J., Nalls, Mike A., Keagle, Pamela, Rivera, Alberto M., Murphy, Natalie A., van Vugt, Joke J.F.A., Geiger, Joshua T., Van der Spek, Rick A., Pliner, Hannah A., Shankaracharya, Smith, Bradley N., Topp, Simon D., Abramzon, Yevgeniya, Gkazi, Athina Soragia, Eicher, John D., Kenna, Aoife, Messina, Sonia, Simone, Isabella L., Ferrucci, Luigi, Moreno, Cristiane de Araujo Martins, Kamalakaran, Sitharthan, Musunuri, Rajeeva Lochan, Evani, Uday Shankar, Abhyankar, Avinash, Zody, Michael C., Wyman, Stacia K., LeNail, Alex, Van Eyk, Jennifer E., Laaksovirta, Hannu, Myllykangas, Liisa, Jansson, Lilja, Valori, Miko, Ealing, John, Hamdalla, Hisham, Rollinson, Sara, Pickering-Brown, Stuart, Orrell, Richard W., Sidle, Katie C., Hardy, John, Singleton, Andrew B., Johnson, Janel O., Arepalli, Sampath, Polak, Meraida, Asress, Seneshaw, Al-Sarraj, Safa, King, Andrew, Troakes, Claire, Vance, Caroline, de Belleroche, Jacqueline, ten Asbroek, Anneloor L.M.A., Muñoz-Blanco, José Luis, Hernandez, Dena G., Ding, Jinhui, Gibbs, J. Raphael, Scholz, Sonja W., Floeter, Mary Kay, Campbell, Roy H., Landi, Francesco, Bowser, Robert, MacGowan, Daniel J.L., Kirby, Janine, Pioro, Erik P., Pamphlett, Roger, Broach, James, Gerhard, Glenn, Dunckley, Travis L., Brady, Christopher B., Kowall, Neil W., Troncoso, Juan C., Le Ber, Isabelle, Heiman-Patterson, Terry D., Kamel, Freya, Van Den Bosch, Ludo, Strom, Tim M., Meitinger, Thomas, Van Eijk, Kristel R., Moisse, Matthieu, McLaughlin, Russell L., Van Es, Michael A., Boylan, Kevin B., Van Blitterswijk, Marka, Morrison, Karen E., Mora, Jesús S., Drory, Vivian E., Shaw, Pamela J., Turner, Martin R., Talbot, Kevin, Fifita, Jennifer A., Nicholson, Garth A., Esteban-Pérez, Jesús, García-Redondo, Alberto, Rogaeva, Ekaterina, Zinman, Lorne, Cooper-Knock, Johnathan, Brice, Alexis, Goutman, Stephen A., Feldman, Eva L., Gibson, Summer B., Van Damme, Philip, Ludolph, Albert C., Andersen, Peter M., Weishaupt, Jochen H., Trojanowski, John Q., Brown, Robert H., Jr., van den Berg, Leonard H., Veldink, Jan H., Stone, David J., Tienari, Pentti, Chiò, Adriano, Shaw, Christopher E., Traynor, Bryan J., and Landers, John E.
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- 2018
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17. Lingual-Alveolar Contact Pressure during Speech in Amyotrophic Lateral Sclerosis: Preliminary Findings
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Searl, Jeff, Knollhoff, Stephanie, and Barohn, Richard J.
- Abstract
Purpose: This preliminary study on lingual-alveolar contact pressures (LACP) in people with amyotrophic lateral sclerosis (ALS) had several aims: (a) to evaluate whether the protocol induced fatigue, (b) to compare LACP during speech (LACP-Sp) and during maximum isometric pressing (LACP-Max) in people with ALS (PALS) versus healthy controls, (c) to compare the percentage of LACP-Max utilized during speech (%Max) for PALS versus controls, and (d) to evaluate relationships between LACP-Sp and LACP-Max with word intelligibility. Method: Thirteen PALS and 12 healthy volunteers produced /t, d, s, z, l, n/ sounds while LACP-Sp was recorded. LACP-Max was obtained before and after the speech protocol. Word intelligibility was obtained from auditory-perceptual judgments. Results: LACP-Max values measured before and after completion of the speech protocol did not differ. LACP-Sp and LACP-Max were statistically lower in the ALS bulbar group compared with controls and PALS with only spinal symptoms. There was no statistical difference between groups for %Max. LACP-Sp and LACP-Max were correlated with word intelligibility. Conclusions: It was feasible to obtain LACP-Sp measures without inducing fatigue. Reductions in LACP-Sp and LACP-Max for bulbar speakers might reflect tongue weakness. Although confirmation of results is needed, the data indicate that individuals with high word intelligibility maintained LACP-Sp at or above 2 kPa and LACP-Max at or above 50 kPa.
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- 2017
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18. NEO1/NEO-EXT studies: Long-term muscle quantitative magnetic resonance imaging and functional efficacy in adults with late-onset Pompe disease (LOPD) on avalglucosidase alfa treatment
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Byrne, Barry, Carlier, Pierre G., Vissing, John, Dimachkie, Mazen M., Barohn, Richard, Kishnani, Priya S., Ladha, Shafeeq, Mengel, Eugen, Sacconi, Sabrina, Trivedi, Jaya, Young, Peter, Haack, Kristina An, Armstrong, Nicole, Miossec, Patrick, Thibault, Nathan, Sparks, Susan, Schoser, Benedikt, and Diaz-Manera, Jordi
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- 2024
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19. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
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Alfano, Lindsay N, Eagle, Michelle, James, Meredith K, Lowes, Linda, Mayhew, Anna, Mazzone, Elena S, Nelson, Leslie, Rose, Kristy J, Abdel-Hamid, Hoda Z, Apkon, Susan D, Barohn, Richard J, Bertini, Enrico, Bloetzer, Clemens, de Vaud, Lausanne Canton, Butterfield, Russell J, Chabrol, Brigitte, Chae, Jong-Hee, Jongno-gu, Daehak-ro, Comi, Giacomi Pietro, Darras, Basil T, Dastgir, Jahannaz, Desguerre, Isabelle, Escobar, Raul G, Finanger, Erika, Guglieri, Michela, Hughes, Imelda, Iannaccone, Susan T, Jones, Kristi J, Karachunski, Peter, Kudr, Martin, Lotze, Timothy, Mah, Jean K, Mathews, Katherine, Nevo, Yoram, Parsons, Julie, Péréon, Yann, de Queiroz Campos Araujo, Alexandra Prufer, Renfroe, J Ben, de Resende, Maria Bernadete Dutra, Ryan, Monique, Selby, Kathryn, Tennekoon, Gihan, Vita, Giuseppe, McDonald, Craig M, Campbell, Craig, Torricelli, Ricardo Erazo, Finkel, Richard S, Flanigan, Kevin M, Goemans, Nathalie, Heydemann, Peter, Kaminska, Anna, Kirschner, Janbernd, Muntoni, Francesco, Osorio, Andrés Nascimento, Schara, Ulrike, Sejersen, Thomas, Shieh, Perry B, Sweeney, H Lee, Topaloglu, Haluk, Tulinius, Már, Vilchez, Juan J, Voit, Thomas, Wong, Brenda, Elfring, Gary, Kroger, Hans, Luo, Xiaohui, McIntosh, Joseph, Ong, Tuyen, Riebling, Peter, Souza, Marcio, Spiegel, Robert J, Peltz, Stuart W, and Mercuri, Eugenio
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- 2017
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20. Minimal manifestation status and prednisone withdrawal in the MGTX trial
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Lee, Ikjae, Kuo, Hui-Chien, Aban, Inmaculada B., Cutter, Gary R., McPherson, Tarrant, Kaminski, Henry J., Sussman, Jon, Ströbel, Philipp, Oger, Joel, Cea, Gabriel, Heckmann, Jeannine M., Evoli, Amelia, Nix, Wilfred, Ciafaloni, Emma, Antonini, Giovanni, Witoonpanich, Rawiphan, King, John O., Beydoun, Said R., Chalk, Colin H., Barboi, Alexandru C., Amato, Anthony A., Shaibani, Aziz I., Katirji, Bashar, Lecky, Bryan R.F., Buckley, Camilla, Vincent, Angela, Dias-Tosta, Elza, Yoshikawa, Hiroaki, Waddington-Cruz, Marcia, Pulley, Michael T., Rivner, Michael H., Kostera-Pruszczyk, Anna, Pascuzzi, Robert M., Jackson, Carlayne E., Verschuuren, Jan J.G., Massey, Janice M., Kissel, John T., Werneck, Lineu C., Benatar, Michael, Barohn, Richard J., Tandan, Rup, Mozaffar, Tahseen, Conwit, Robin, Minisman, Greg, Sonett, Joshua R., and Wolfe, Gil I.
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- 2020
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21. Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS
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Benatar, Michael, Zhang, Lanyu, Wang, Lily, Granit, Volkan, Statland, Jeffrey, Barohn, Richard, Swenson, Andrea, Ravits, John, Jackson, Carlayne, Burns, Ted M., Trivedi, Jaya, Pioro, Erik P., Caress, James, Katz, Jonathan, McCauley, Jacob L., Rademakers, Rosa, Malaspina, Andrea, Ostrow, Lyle W., and Wuu, Joanne
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- 2020
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22. Practice advisory: Thymectomy for myasthenia gravis (practice parameter update): Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology
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Gronseth, Gary S., Barohn, Richard, and Narayanaswami, Pushpa
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- 2020
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23. Episodic Ataxia Type 1: Natural History and Effect on Quality of Life.
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Graves, Tracey D., Griggs, Robert C., Bundy, Brian N., Jen, Joanna C., Baloh, Robert W., Hanna, Michael G., the CINCH Investigators, Jen, Joanna J., Amato, Anthony A., Barohn, Richard J., Hahn, Angelika F., Hart, Kimberly, Herr, Barbara, Wang, Yunxia, Salajegheh, Mohammad, Puwanant, Araya, Rajakalendran, Sanjeev, Cha, Yoon-Hee, Krischer, Jeffrey, and Herbelin, Laura
- Subjects
INTERACTIVE voice response (Telecommunication) ,NATURAL history ,SPINOCEREBELLAR ataxia ,CLUSTER headache ,ATAXIA ,QUALITY of life - Abstract
Episodic ataxia type 1 (EA1) is a rare autosomal potassium channelopathy, due to mutations in KCNA1. Patients have childhood onset of intermittent attacks of ataxia, dizziness or imbalance. In order to quantify the natural history of EA1, its effect on quality of life and in preparation for future clinical trials, we set up an international multi-centre study of EA1. We recruited thirty-three participants with EA1: twenty-three completed 1-year follow-up and eighteen completed 2-year follow-up. There was very little accumulation of disability or impairment over the course of the 2 years of the study. The outcome measures of ataxia (SARA and functional rating of ataxia) and the activities of daily living scale were largely stable over time. Self-reported health-related quality of life (SF-36) scores were lower across all domains than controls, in keeping with a chronic condition. Physical subdomain scores appeared to deteriorate over time, which seems to be driven by the female participants in the study. This is an interesting finding and warrants further study. Attacks of EA1 reported by participants in real time via an interactive voice response system showed that symptoms were not stereotyped; however, attack duration and frequency was stable between individuals. This large prospective study is the first ever completed in subjects with EA1. We document the natural history of the disorder over 2 years. These data will enable the development of outcome measures for clinical trials of treatment. [ABSTRACT FROM AUTHOR]
- Published
- 2023
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24. Using automated electronic medical record data extraction to model ALS survival and progression
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Karanevich, Alex G., Weisbrod, Luke J., Jawdat, Omar, Barohn, Richard J., Gajewski, Byron J., He, Jianghua, and Statland, Jeffrey M.
- Published
- 2018
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25. Satisfactory Response With Achieving Maintenance Low-Dose Prednisone in Generalized Myasthenia Gravis
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Abuzinadah, Ahmad R., Jabari, Duaa, Jawdat, Omar, Pasnoor, Mamatha, Glenn, Melanie, Herbelin, Laura, McVey, April L., Barohn, Richard J., and Dimachkie, Mazen M.
- Published
- 2018
- Full Text
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26. Disease course and therapeutic approach in dermatomyositis: A four-center retrospective study of 100 patients
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Johnson, Nicholas E., Arnold, W. David, Hebert, Donald, Gwathmey, Kelly, Dimachkie, Mazen M., Barohn, Richard J., McVey, April L., Pasnoor, Mamatha, Amato, Anthony A., McDermott, Michael P., Kissel, John, and Heatwole, Chad R.
- Published
- 2015
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27. Team Science and Advancing Research at the University of Missouri
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Barohn, Richard J.
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Medical School Perspective - Published
- 2023
28. Review process for IVIg treatment: Lessons learned from INSIGHTS neuropathy study
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Levine, Todd D., Katz, Jonathan S., Barohn, Richard, Vaughan, Leslie J., Dimachkie, Mazen M., Saperstein, David S., Mozaffar, Tahseen, Wolfe, Gil I., Mayo, Matthew S., Badger, Gary J., Katzin, Lara, Ritt, Elissa, Greer, Michelle, DiStefano, Joseph, and Schmidt, Patrick M.
- Published
- 2018
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29. Toxic myopathies
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Pasnoor, Mamatha, Barohn, Richard J., and Dimachkie, Mazen M.
- Published
- 2018
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30. An instrumented timed up and go in facioscapulohumeral muscular dystrophy
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Huisinga, Jessie, Bruetsch, Adam, Mccalley, Ayla, Currence, Melissa, Herbelin, Laura, Jawdat, Omar, Pasnoor, Mamatha, Dimachkie, Mazen, Barohn, Richard, and Statland, Jeffrey
- Published
- 2018
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31. Learning from the past: reflections on recently completed myasthenia gravis trials
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Benatar, Michael, Howard, James F., Jr., Barohn, Richard, Wolfe, Gil I., and Cutter, Gary
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- 2018
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32. 2017 European League Against Rheumatism/American College of Rheumatology classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups
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Lundberg, Ingrid E, Tjärnlund, Anna, Bottai, Matteo, Werth, Victoria P, Pilkington, Clarissa, Visser, Marianne de, Alfredsson, Lars, Amato, Anthony A, Barohn, Richard J, Liang, Matthew H, Singh, Jasvinder A, Aggarwal, Rohit, Arnardottir, Snjolaug, Chinoy, Hector, Cooper, Robert G, Dankó, Katalin, Dimachkie, Mazen M, Feldman, Brian M, Torre, Ignacio Garcia-De La, Gordon, Patrick, Hayashi, Taichi, Katz, James D, Kohsaka, Hitoshi, Lachenbruch, Peter A, Lang, Bianca A, Li, Yuhui, Oddis, Chester V, Olesinska, Marzena, Reed, Ann M, Rutkowska-Sak, Lidia, Sanner, Helga, Selva-O’Callaghan, Albert, Song, Yeong-Wook, Vencovsky, Jiri, Ytterberg, Steven R, Miller, Frederick W, and Rider, Lisa G
- Published
- 2017
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33. Using a Bayesian model of the joint distribution of pain and time on medication to decide on pain medication for neuropathy.
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Gao, Guangyi, Wick, Jo A., Brown, Alexandra R., Barohn, Richard J., and Gajewski, Byron J.
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JOINT pain ,PANEL analysis ,DRUGS ,NEUROPATHY ,PAIN management - Abstract
The PAIN-CONTRoLS trial compared four medications in treating Cryptogenic sensory polyneuropathy. The primary outcome was a utility function that combined two outcomes, patients' pain score reduction and patients' quit rate. However, additional analysis of the individual outcomes could also be leveraged to inform selecting an optimal medication for future patients. We demonstrate how joint modeling of longitudinal and time-to-event data from PAIN-CONTRoLS can be used to predict the effects of medication in a patient-specific manner and helps to make patient-focused decisions. A joint model was used to evaluate the two outcomes while accounting for the association between the longitudinal process and the time-to-event processes. Results suggested no significant association between the patients' pain scores and time to the medication quit in the PAIN-CONTRoLS study, but the joint model still provided robust estimates and a better model fit. Using the model estimates, given patients' baseline characteristics, a drug profile on both the pain reduction and medication time could be obtained for each drug, providing information on how likely they would quit and how much pain reduction they should expect. Our analysis suggested that drugs viable for one patient may not be beneficial for others. [ABSTRACT FROM AUTHOR]
- Published
- 2023
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34. Phase 2 trial in acetylcholine receptor antibody‐positive myasthenia gravis of transition from intravenous to subcutaneous immunoglobulin: The MGSCIg study.
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Pasnoor, Mamatha, Bril, Vera, Levine, Todd, Trivedi, Jaya, Silvestri, Nicholas J., Phadnis, Milind, Katzberg, Hans D., Saperstein, David S., Wolfe, Gil I., Herbelin, Laura, Higgs, Kiley, Heim, Andrew J., Statland, Jeffrey M., Barohn, Richard J., and Dimachkie, Mazen M.
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MYASTHENIA gravis ,CHOLINERGIC receptors ,PATIENT satisfaction ,CLINICAL deterioration ,CLINICAL medicine ,INTRAVENOUS immunoglobulins - Abstract
Background and purpose: Data on maintenance therapy with subcutaneous immunoglobulin (SCIg) in myasthenia gravis (MG) are limited. We report on transitioning acetylcholine receptor (AChR) antibody‐positive (Ab+) MG patients on stable intravenous immunoglobulin (IVIg) regimens as part of routine clinical care to SCIg 1:1.2. Methods: This multicenter North American open‐label prospective investigator‐initiated study had two components: the IVIg Stabilization Period (ISP) enrolling patients already on IVIg as part of routine clinical care (Weeks −10 to −1), followed by transition of stable MG subjects to SCIg in the Experimental Treatment Period (ETP; Weeks 0 to 12). We hypothesized that >65% of patients entering the ETP would have a stable Quantitative Myasthenia Gravis (QMG) score from Week 0 to Week 12. Secondary outcome measures included other efficacy measures, safety, tolerability, IgG levels, and treatment satisfaction. Results: We recruited 23 patients in the ISP, and 22 entered the ETP. A total of 12 subjects (54.5%) were female, and 18 (81.8%) were White, with mean age 51.4 ± 17 years. We obtained Week 12 ETP QMG data on 19 of 22; one subject withdrew from ETP owing to clinical deterioration, and two subjects withdrew due to dislike of needles. On primary analysis, 19 of 22 participants (86.4%, 95% confidence interval = 0.72–1.00) were treatment successes using last observation carried forward (p = 0.018). Secondary efficacy measures supported MG stability. SCIg was safe and well tolerated, and IgG levels were stable. Treatment satisfaction was comparable between ISP and ETP. Conclusions: MG patients on IVIg as part of their routine clinical care remained stable on monthly IVIg dosage, and most maintained similar disease stability on SCIg. [ABSTRACT FROM AUTHOR]
- Published
- 2023
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35. Congenital Myasthenic Syndromes: a Clinical and Treatment Approach
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Farmakidis, Constantine, Pasnoor, Mamatha, Barohn, Richard J., and Dimachkie, Mazen M.
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- 2018
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36. Update on Inclusion Body Myositis
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Jabari, Duaa, Vedanarayanan, V. V., Barohn, Richard J., and Dimachkie, Mazen M.
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- 2018
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37. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
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Crow, Rebecca A., Hart, Kimberly A., McDermott, Michael P., Tawil, Rabi, Martens, William B., Herr, Barbara E., McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M., Eagle, Michele, Brown, Mary W., Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan Y., Morandi, Lucia, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Flanigan, Kevin M., Kuntz, Nancy L., Mah, Jean K., Morrison, Leslie, Darras, Basil T., von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, McDonald, Craig M., Vita, Giuseppe, Barohn, Richard J., Finkel, Richard S., Wicklund, Matthew, McMillan, Jr, Hugh J., Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W., Howard, James F., Thangarajh, Mathula, Campbell, Craig, Griggs, Robert C., Bushby, Kate, and Guglieri, Michela
- Published
- 2018
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38. Activity for Diabetic Polyneuropathy (ADAPT): study design and protocol for a 2-site randomized controlled trial
- Author
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Kluding, Patricia M., Singleton, J. Robinson, Pasnoor, Mamatha, Dimachkie, Mazen M., Barohn, Richard J., Smith, A. Cordon, and Marcus, Robin L.
- Subjects
Lifestyles -- Health aspects ,Quality of life -- Health aspects ,Diabetic neuropathies -- Development and progression -- Care and treatment ,Health - Abstract
Background. Half of all patients with diabetes develop diabetic peripheral neuropathy (DPN), a complication leading to reduced mobility and quality of life. Although there are no proven pharmacologic approaches to reduce DPN risk or slow its progression, evidence suggests that physical activity may improve symptoms and enhance peripheral nerve regeneration. Objective. The aim of the study will be to determine the impact of an intense lifestyle intervention on neuropathy progression and quality of life in individuals with DPN. Design. The study will be a randomized controlled trial. Setting. The study will be conducted at 2 academic medical centers. Participants. The participants will be 140 individuals with type 2 diabetes and mild to moderate DPN. Intervention. The intervention group will receive 18 months of supervised exercise training, actigraphy-based counseling to reduce sedentary behavior, and individualized dietary counseling. Control group participants will receive diet and activity counseling at baseline and at 9 months. Measurements. The primary outcomes are neuropathy progression as measured by intraepidermal nerve fiber density in a distal thigh skin biopsy and the Norfolk Quality of Life-Diabetic Neuropathy score. Secondary outcomes include pain, gait, balance, and mobility measures. Limitations. Due to the combined intervention approach, this protocol will not be able to determine which intervention components influence outcomes. There also may be difficulty with participant attrition during the 18-month study intervention. Conclusions. The Activity for Diabetic Polyneuropathy (ADAPT) protocol resulted from a collaboration between physical therapists and neurologist researchers that includes as primary outcomes both a quality-of-life measure (NQOL-DN) and a physiologic biomarker (IENFD). It has the potential to demonstrate that an intensive lifestyle intervention may be a sustainable, clinically effective approach for people with DPN that improves patient outcomes and can have an immediate impact on patient care and future clinical trials., Approximately 30 million Americans have diabetes, half of whom will develop diabetic peripheral neuropathy (DPN). (1) Diabetic peripheral neuropathy causes reduced mobility and quality of life due to pain, sensory [...]
- Published
- 2017
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39. High frequency of gastrointestinal manifestations in myotonic dystrophy type 1 and type 2
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Hilbert, James E., Barohn, Richard J., Clemens, Paula R., Luebbe, Elizabeth A., Martens, William B., McDermott, Michael P., Parkhill, Amy L., Tawil, Rabi, Thornton, Charles A., and Moxley, Richard T., III
- Published
- 2017
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40. Less is More in Diabetic Neuropathy Diagnosis: Comparison of Quantitative Sudomotor Axon Reflex and Skin Biopsy
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Abuzinadah, Ahmad R., Kluding, Patricia, Wright, Douglas, DʼSilva, Linda, Ryals, Janelle, Hendry, Bill, Jawdat, Omar, Herbelin, Laura, McVey, April L., Barohn, Richard J., Dimachkie, Mazen M., and Pasnoor, Mamatha
- Published
- 2017
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41. EFFICACY OF PREDNISONE FOR THE TREATMENT OF OCULAR MYASTHENIA (EPITOME): A RANDOMIZED, CONTROLLED TRIAL
- Author
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BENATAR, MICHAEL, MCDERMOTT, MICHAEL P., SANDERS, DONALD B., WOLFE, GIL I., BAROHN, RICHARD J., NOWAK, RICHARD J., HEHIR, MICHAEL, JUEL, VERN, KATZBERG, HANS, and TAWIL, RABI
- Published
- 2016
- Full Text
- View/download PDF
42. Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine
- Author
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Burns, Ted M., Smith, Gordon A., Allen, Jeffrey A., Amato, Anthony A., Arnold, David W., Barohn, Richard, Benatar, Michael, Bird, Shawn J., Bromberg, Mark, Chahin, Nizar, Ciafaloni, Emma, Cohen, Jeffrey A., Corse, Andrea, Crum, Brian A., David, William S., Dimberg, Elliot, Sousa, Eduardo A. De, Donofrio, Peter D., Dyck, James P. B., Engel, Andrew G., Ensrud, Erik R., Ferrante, Mark, Freimer, Miriam, Gable, Karissa L., Gibson, Summer, Gilchrist, James M., Goldstein, Jonathan M., Gooch, Clifton L., Goodman, Brent P., Gorelov, Dmitri, Gospe, Sidney M., Jr., Goyal, Namita A., Guidon, Amanda C., Guptill, Jeffrey T., Gutmann, Laurie, Gutmann, Ludwig, Gwathmey, Kelly, Harati, Yadollah, Harper, Michel C., Jr., Hehir, Michael K., Hobson-Webb, Lisa D., Howard, James F., Jr., Jackson, Carlayne E., Johnson, Nicholas, Jones, Sarah M., Juel, Vern C., Kaminski, Henry J., Karam, Chafic, Kennelly, Kathleen D., Khella, Sami, Khoury, Julie, Kincaid, John C., Kissel, John T., Kolb, Noah, Lacomis, David, Ladha, Shafeeq, Larriviere, Daniel, Lewis, Richard A., Li, Yuebing, Litchy, William J., Logigian, Eric, Lou, Jau-Shin, MacGowen, Daniel J.L., Maselli, Ricardo, Massey, Janice M., Mauermann, Michelle L., Mathews, Katherine D., Meriggioli, Matthew N., Miller, Robert G., Moon, Joon-Shik, Mozaffar, Tahseen, Nations, Sharon P., Nowak, Richard J., Ostrow, Lyle W., Pascuzzi, Robert M., Peltier, Amanda, Ruzhansky, Katherine, Richman, David P., Ross, Mark A., Rubin, DEVON I., Russell, James A., Sachs, George M., Salajegheh, Mohammad Kian, Saperstein, David S., Scelsa, Stephen, Selcen, Duygu, Shaibani, Aziz, Shieh, Perry B., Silvestri, Nicholas J., Singleton, Rob J., Smith, Benn E., So, Yuen T., Solorzano, Guillermo, Sorenson, Eric J., Srinivasen, Jayashri, Tavee, Jinny, Tawil, Rabi, Thaisetthawatkul, Pariwat, Thornton, Charles, Trivedi, Jaya, Vernino, Steven, Wang, Annabel K., Webb, Tyler A., Weiss, Michael D., Windebank, Anthony J., and Wolfe, Gil I.
- Published
- 2016
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43. Rare variants in SQSTM1 and VCP genes and risk of sporadic inclusion body myositis
- Author
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Hanna, Michael G., Houlden, Henry, Machado, Pedro M., Gang, Qiang, Bettencourt, Conceicao, Healy, Estelle, Parton, Matthew, Holton, Janice L., Brady, Stefen, Hilton-Jones, David, Shieh, Perry B., Zanoteli, Edmar, de Camargo, Leonardo Valente, De Paepe, Boel, De Bleecker, Jan, Shaibani, Aziz, Ripolone, Michela, Violano, Raffaella, Moggio, Maurizio, Barohn, Richard J., Dimachkie, Mazen M., McVey, April L., Pasnoor, Mamatha, Glenn, Melanie, Jawdat, Omar, Statland, Jeffrey, Rico, Gabrielle, Mora, Marina, Mantegazza, Renato, Zanotti, Simona, Needham, Merrilee, Mastaglia, Frank, Liang, Christina, Dalakas, Marinos C., Biba, Angie, Chinoy, Hector, Lilleker, James B., Lamb, Janine, Platt, Hazel, Cooper, Robert G., Miller, James A.L., Roberts, Mark, Househam, Elizabeth, Hilton, David, Shivane, Aditya, Bartlett, Amy, Kissel, John T., Runk, Heidi, Wicklund, Matthew, Saperstein, David S., McKinney, Lynette R., Bettencourt, Conceição, Pittman, Alan M., Hughes, Deborah, and Singleton, Andrew B.
- Published
- 2016
- Full Text
- View/download PDF
44. New insights into B cell and T cell contributions to myasthenia gravis immune dysregulation: 31
- Author
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Cao, Yonghao, Lee, Jae-Yun, Stathopoulos, Panos, Gupta, Sasha, Amezquita, Robert A., Kleinstein, Steven H., Goldstein, Jonathan M., Barohn, Richard J., Dimachkie, Mazen M., Nowak, Richard J., and OʼConnor, Kevin C.
- Published
- 2015
45. A Genome-Wide Association Study of Myasthenia Gravis
- Author
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Renton, Alan E., Pliner, Hannah A., Provenzano, Carlo, Evoli, Amelia, Ricciardi, Roberta, Nalls, Michael A., Marangi, Giuseppe, Abramzon, Yevgeniya, Arepalli, Sampath, Chong, Sean, Hernandez, Dena G., Johnson, Janel O., Bartoccioni, Emanuela, Scuderi, Flavia, Maestri, Michelangelo, Gibbs, J. Raphael, Errichiello, Edoardo, Chiò, Adriano, Restagno, Gabriella, Sabatelli, Mario, Macek, Mark, Scholz, Sonja W., Corse, Andrea, Chaudhry, Vinay, Benatar, Michael, Barohn, Richard J., McVey, April, Pasnoor, Mamatha, Dimachkie, Mazen M., Rowin, Julie, Kissel, John, Freimer, Miriam, Kaminski, Henry J., Sanders, Donald B., Lipscomb, Bernadette, Massey, Janice M., Chopra, Manisha, Howard, James F., Jr, Koopman, Wilma J., Nicolle, Michael W., Pascuzzi, Robert M., Pestronk, Alan, Wulf, Charlie, Florence, Julaine, Blackmore, Derrick, Soloway, Aimee, Siddiqi, Zaeem, Muppidi, Srikanth, Wolfe, Gil, Richman, David, Mezei, Michelle M., Jiwa, Theresa, Oger, Joel, Drachman, Daniel B., and Traynor, Bryan J.
- Published
- 2015
- Full Text
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46. Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT
- Author
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Amato, Anthony A, Hanna, Michael G, Machado, Pedro M, Badrising, Umesh A, Chinoy, Hector, Benveniste, Olivier, Karanam, Ananda Krishna, Wu, Min, Tankó, László B, Schubert-Tennigkeit, Agnes Annette, Papanicolaou, Dimitris A, Lloyd, Thomas E, Needham, Merrilee, Liang, Christina, Reardon, Katrina A, de Visser, Marianne, Ascherman, Dana P, Barohn, Richard J, Dimachkie, Mazen M, Miller, James A L, Kissel, John T, Oskarsson, Björn, Joyce, Nanette C, Van den Bergh, Peter, Baets, Jonathan, De Bleecker, Jan L, Karam, Chafic, David, William S, Mirabella, Massimiliano, Nations, Sharon P, et al, and University of Zurich
- Subjects
2728 Neurology (clinical) ,610 Medicine & health ,10040 Clinic for Neurology - Published
- 2021
47. A secondary analysis of PAIN‐CONTRoLS: Pain's impact on sleep, fatigue, and activities of daily living.
- Author
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Bhai, Salman F., Brown, Alexandra, Gajewski, Byron, Kimminau, Kim S., Waitman, Lemuel R., Pasnoor, Mamatha, and Barohn, Richard J.
- Abstract
Introduction/Aims: Peripheral neuropathies commonly affect quality of life of patients due to pain, sleep disturbances, and fatigue, although trials have not adequately explored these domains of care. The aim of this study was to assess the impact of nortriptyline, duloxetine, pregabalin, and mexiletine on pain, sleep, and fatigue in patients diagnosed with cryptogenic sensory polyneuropathy (CSPN). Methods: We implemented a Bayesian adaptive design to perform a 12‐wk multisite, randomized, prospective, open‐label comparative effectiveness study in 402 CSPN patients. Participants received either nortriptyline (n = 134), duloxetine (n = 126), pregabalin (n = 73), or mexiletine (n = 69). At prespecified analysis timepoints, secondary outcomes, Patient Reported Outcomes Measurement Information System (PROMIS) surveys including Short Form (SF)‐12, pain interference, fatigue, and sleep disturbance, were collected. Results: Mexiletine had the highest quit rate (58%) due to gastrointestinal side effects, while nortriptyline (38%) and duloxetine (38%) had the lowest quit rates. If tolerated for the full 12 wk of the study, mexiletine had the highest probability (>90%) of positive outcomes for improvements in pain interference and fatigue. There was no significant difference among the medications for sleep disturbance or SF‐12 scores. Adverse events and lack of efficacy were the two most common reasons for cessation of therapy. Discussion: Physicians caring for patients with CSPN should consider mexiletine to address pain and fatigue, although nortriptyline and duloxetine are better medications to trial first since they are better tolerated. Future research should compare other commonly used medications for CSPN to determine evidence‐based treatment strategies. See Editorial on pages 380‐381 in this issue [ABSTRACT FROM AUTHOR]
- Published
- 2022
- Full Text
- View/download PDF
48. Chronic Inflammatory Demyelinating Polyneuropathy
- Author
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Dimachkie, Mazen M. and Barohn, Richard J.
- Published
- 2013
- Full Text
- View/download PDF
49. Guillain-Barré Syndrome
- Author
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Dimachkie, Mazen M. and Barohn, Richard J.
- Published
- 2013
- Full Text
- View/download PDF
50. Inclusion Body Myositis
- Author
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Dimachkie, Mazen M. and Barohn, Richard J.
- Published
- 2013
- Full Text
- View/download PDF
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