Search

Your search keyword '"McDonald, Craig M."' showing total 723 results
Start Over Author "McDonald, Craig M."
723 results on '"McDonald, Craig M."'

53. Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy(1)

Author

McDonald, Craig M, Mayer, Oscar H, Hor, Kan N, Miller, Debra, Goemans, Nathalie, Henricson, Erik K, Marden, Jessica R, Freimark, Jonathan, Lane, Henry, Zhang, Adina, Frean, Molly, Trifillis, Panayiota, Koladicz, Karyn, and Signorovitch, James

Subjects
Duchenne muscular dystrophy, PREDNISONE, BOYS, Science & Technology, ambulation, Clinical Neurology, Neurosciences, DEFLAZACORT, BENEFITS, MANAGEMENT, SURVIVAL, TRIAL, Neurosciences & Neurology, GLUCOCORTICOIDS, Treatment outcome, Life Sciences & Biomedicine
Abstract

BACKGROUND: Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is limited. OBJECTIVE: Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambulatory boys with DMD. METHODS: Disease progression was measured by functional status (Performance of Upper Limb Module for DMD 1.2 [PUL] and Egen Klassifikation Scale Version 2 [EK] scale) and by cardiac and pulmonary function (left ventricular ejection fraction [LVEF], forced vital capacity [FVC] % -predicted, cough peak flow [CPF]). Longitudinal changes in outcomes, progression to key disease milestones, and dosing and body composition metrics were analyzed descriptively and in multivariate models. RESULTS: This longitudinal cohort study included 86 non-ambulatory patients with DMD (mean age 13.4 years; n = 40 [deflazacort], n = 29 [prednisone], n = 17 [no steroids]). Deflazacort use resulted in slower average declines in FVC % -predicted vs. no steroids (+3.73 percentage points/year, p

Published
2023

54. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

Author

Guglieri, Michela, primary, Clemens, Paula R., additional, Perlman, Seth J., additional, Smith, Edward C., additional, Horrocks, Iain, additional, Finkel, Richard S., additional, Mah, Jean K., additional, Deconinck, Nicolas, additional, Goemans, Nathalie, additional, Haberlova, Jana, additional, Straub, Volker, additional, Mengle-Gaw, Laurel J., additional, Schwartz, Benjamin D., additional, Harper, Amy D., additional, Shieh, Perry B., additional, De Waele, Liesbeth, additional, Castro, Diana, additional, Yang, Michelle L., additional, Ryan, Monique M., additional, McDonald, Craig M., additional, Tulinius, Mar, additional, Webster, Richard, additional, McMillan, Hugh J., additional, Kuntz, Nancy L., additional, Rao, Vashmi K., additional, Baranello, Giovanni, additional, Spinty, Stefan, additional, Childs, Anne-Marie, additional, Sbrocchi, Annie M., additional, Selby, Kathryn A., additional, Monduy, Migvis, additional, Nevo, Yoram, additional, Vilchez-Padilla, Juan J., additional, Nascimento-Osorio, Andres, additional, Niks, Erik H., additional, de Groot, Imelda J.M., additional, Katsalouli, Marina, additional, James, Meredith K., additional, van den Anker, Johannes, additional, Damsker, Jesse M., additional, Ahmet, Alexandra, additional, Ward, Leanne M., additional, Jaros, Mark, additional, Shale, Phil, additional, Dang, Utkarsh J., additional, and Hoffman, Eric P., additional

Published
2022
Full Text
View/download PDF

56. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial

Author

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
Full Text
View/download PDF

57. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

Author

Muntoni, Francesco, primary, Guglieri, Michela, additional, Mah, Jean K., additional, Wagner, Kathryn R., additional, Brandsema, John F., additional, Butterfield, Russell J., additional, McDonald, Craig M., additional, Mayhew, Anna G., additional, Palmer, Jeffrey P., additional, Marraffino, Shannon, additional, Charnas, Lawrence, additional, and Mercuri, Eugenio, additional

Published
2022
Full Text
View/download PDF

59. Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

Author

Connolly, Anne M., Florence, Julaine M., Cradock, Mary M., Malkus, Elizabeth C., Schierbecker, Jeanine R., Siener, Catherine A., Wulf, Charlie O., Anand, Pallavi, Golumbek, Paul T., Zaidman, Craig M., Philip Miller, J., Lowes, Linda P., Alfano, Lindsay N., Viollet-Callendret, Laurence, Flanigan, Kevin M., Mendell, Jerry R., McDonald, Craig M., Goude, Erica, Johnson, Linda, Nicorici, Alina, Karachunski, Peter I., Day, John W., Dalton, Joline C., Farber, Janey M., Buser, Karen K., Darras, Basil T., Kang, Peter B., Riley, Susan O., Shriber, Elizabeth, Parad, Rebecca, Bushby, Kate, and Eagle, Michelle

Published
2013
Full Text
View/download PDF

71. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Author

McDonald, Craig M, primary, Marbán, Eduardo, additional, Hendrix, Suzanne, additional, Hogan, Nathaniel, additional, Ruckdeschel Smith, Rachel, additional, Eagle, Michelle, additional, Finkel, Richard S, additional, Tian, Cuixia, additional, Janas, Joanne, additional, Harmelink, Matthew M, additional, Varadhachary, Arun S, additional, Taylor, Michael D, additional, Hor, Kan N, additional, Mayer, Oscar H, additional, Henricson, Erik K, additional, Furlong, Pat, additional, Ascheim, Deborah D, additional, Rogy, Siegfried, additional, Williams, Paula, additional, Marbán, Linda, additional, Butterfield, Russell, additional, Connolly, Anne, additional, Muntoni, Francesco, additional, Joyce, Nanette C., additional, Evans, Maya, additional, Abedi, Mehrdad, additional, Surampudi, Prasanth, additional, Jhawar, Sanjay, additional, Dayan, Jonathan G., additional, Anthonisen, Colleen, additional, Goude, Erica, additional, Nicorici, Alina, additional, Sarwary, Omaid, additional, Prasad, Poonam, additional, Baek, Jayoon, additional, Newton, Andrew, additional, Johnson, Hannah, additional, Kusmik, Kyle, additional, Filar, Lauri, additional, Edmondson, Angie, additional, Rybalsky, Irina, additional, Chouteau, Wendy, additional, Giordano, Anthony F., additional, Rodriguez, Aixa, additional, Anderson, Kristan, additional, Wezel, Germaine, additional, Vega, Melisa, additional, Duke, Julie, additional, Collado, Jorge, additional, Civitello, Matthew, additional, Wells, Julie, additional, Pyzik, Erika, additional, Rehborg, Rebecca, additional, Brown, Michelle, additional, Van Eyk, Jennifer, additional, and Rogers, Russell G., additional

Published
2022
Full Text
View/download PDF

72. Additional file 4 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Abstract

Additional file 4: Peak and global mid ventricular strain (εcc %) in unaffected controls (n=15) and individuals with DMD (n=46) at baseline (UF Cohort).

Published
2022
Full Text
View/download PDF

73. Additional file 2 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Abstract

Additional file 2: Longitudinal changes in global strain in DMD. Solid line for global strain was defined based on normal zone cut off of -17% as given by HARP software. Red lines indicates subjects with more than 5 years data, filled triangles represent unaffected controls.

Published
2022
Full Text
View/download PDF

74. Additional file 6 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 6: Comparison of cardiac function in control and DMD subjects at baseline (UF Cohort).

Published
2022
Full Text
View/download PDF

75. sj-docx-1-cic-10.1177_11795468221116838 – Supplemental material for Influence of β1 Adrenergic Receptor Genotype on Longitudinal Measures of Left Ventricular Ejection Fraction and Responsiveness to ß-Blocker Therapy in Patients With Duchenne Muscular Dystrophy

Author

Kelley, Eli F, Cross, Troy J, McDonald, Craig M, Investigators, CINRG, Hoffman, Eric P, Spurney, Christopher F, and Bello, Luca

Subjects
Cardiology
Abstract

Supplemental material, sj-docx-1-cic-10.1177_11795468221116838 for Influence of β1 Adrenergic Receptor Genotype on Longitudinal Measures of Left Ventricular Ejection Fraction and Responsiveness to ß-Blocker Therapy in Patients With Duchenne Muscular Dystrophy by Eli F Kelley, Troy J Cross, Craig M McDonald, CINRG Investigators, Eric P Hoffman, Christopher F Spurney and Luca Bello in Clinical Medicine Insights: Cardiology

Published
2022
Full Text
View/download PDF

76. Additional file 3 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities, sense organs, skin and connective tissue diseases, nervous system diseases
Abstract

Additional file 3: Longitudinal change in global strain for mid ventricle of DMD.

Published
2022
Full Text
View/download PDF

77. Additional file 5 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 5: Strain for each LV segment in controls and individuals with DMD (n=46) at baseline (UF cohort).

Published
2022
Full Text
View/download PDF

78. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy

Author

Mah, Jean K., Clemens, Paula R., Guglieri, Michela, Smith, Edward C., Finkel, Richard S., Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Castro, Diana, Kuntz, Nancy L., McDonald, Craig M., Damsker, Jesse M., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Jackowski, Stefan, Stimpson, Georgia, Ridout, Deborah A., Ayyar-Gupta, Vandana, Baranello, Giovanni, Manzur, Adnan Y., Muntoni, Francesco, Gordish-Dressman, Heather, Leinonen, Mika, Ward, Leanne M., Hoffman, Eric P., and Dang, Utkarsh J.

Subjects
Male, Research, Anti-Inflammatory Agents, Body Height, United Kingdom, Muscular Dystrophy, Duchenne, Online Only, Treatment Outcome, Neurology, Child, Preschool, Humans, Muscle Strength, Child, Glucocorticoids, Pregnadienediols, Original Investigation
Abstract

This nonrandomized controlled trial examines efficacy of vamorolone treatment for Duchenne muscular dystrophy among boys compared with glucocorticoid treatment., Key Points Question Is long-term treatment with vamorolone associated with sustained disease modification and decrease of glucocorticoid-associated adverse outcomes among patients with Duchenne muscular dystrophy (DMD)? Findings In this nonrandomized controlled trial of 46 boys with DMD, higher-dose vamorolone treatment for up to 30 months was not associated with a change in time to stand velocity. Vamorolone was associated with continued disease-modification and similar efficacy compared with glucocorticoid treatment from 2 historical control cohorts, and long-term vamorolone treatment was associated with decreased adverse outcomes compared with traditional glucocorticoid therapy. Meaning These findings suggest that vamorolone may have potential as a standard of care disease-modifying treatment for boys with DMD., Importance Vamorolone is a synthetic steroidal drug with potent anti-inflammatory properties. Initial open-label, multiple ascending dose-finding studies of vamorolone among boys with Duchenne muscular dystrophy (DMD) found significant motor function improvement after 6 months treatment in higher-dose (ie, ≥2.0 mg/kg/d) groups. Objective To investigate outcomes after 30 months of open-label vamorolone treatment. Design, Setting, and Participants This nonrandomized controlled trial was conducted by the Cooperative International Neuromuscular Research Group at 11 US and non-US study sites. Participants were 46 boys ages 4.5 to 7.5 years with DMD who completed the 6-month dose-finding study. Data were analyzed from July 2020 through November 2021. Interventions Participants were enrolled in a 24-month, long-term extension (LTE) study with vamorolone dose escalated to 2.0 or 6.0 mg/kg/d. Main Outcomes and Measures Change in time-to-stand (TTSTAND) velocity from dose-finding baseline to end of LTE study was the primary outcome. Efficacy assessments included timed function tests, 6-minute walk test, and NorthStar Ambulatory Assessment (NSAA). Participants with DMD treated with glucocorticoids from the Duchenne Natural History Study (DNHS) and NorthStar United Kingdom (NSUK) Network were matched and compared with participants in the LTE study receiving higher doses of vamorolone. Results Among 46 boys with DMD who completed the dose-finding study, 41 boys (mean [SD] age, 5.33 [0.96] years) completed the LTE study. Among 21 participants treated with higher-dose (ie, ≥2.0 mg/kg/d) vamorolone consistently throughout the 6-month dose-finding and 24-month LTE studies with data available at 30 months, there was a decrease in mean (SD) TTSTAND velocity from baseline to 30 months (0.206 [0.070] rises/s vs 0.189 (0.124) rises/s), which was not a statistically significant change (−0.011 rises/s; CI, −0.068 to 0.046 rises/s). There were no statistically significant differences between participants receiving higher-dose vamorolone and matched participants in the historical control groups receiving glucocorticoid treatment (75 patients in DNHS and 110 patients in NSUK) over a 2-year period in NSAA total score change (0.22 units vs NSUK; 95% CI, −4.48 to 4.04]; P = .92), body mass index z score change (0.002 vs DNHS SD/mo; 95% CI, −0.006 to 0.010; P = .58), or timed function test change. Vamorolone at doses up to 6.0 mg/kg/d was well tolerated, with 5 of 46 participants discontinuing prematurely and for reasons not associated with study drug. Participants in the DNHS treated with glucocorticoids had significant growth delay in comparison with participants treated with vamorolone who had stable height percentiles (0.37 percentile/mo; 95% CI, 0.23 to 0.52 percentile/mo) over time. Conclusions and Relevance This study found that vamorolone treatment was not associated with a change in TTSTAND velocity from baseline to 30 months among boys with DMD aged 4 to 7 years at enrollment. Vamorolone was associated with maintenance of muscle strength and function up to 30 months, similar to standard of care glucocorticoid therapy, and improved height velocity compared with growth deceleration associated with glucocorticoid treatment, suggesting that vamorolone may be an attractive candidate for treatment of DMD. Trial Registration ClinicalTrials.gov Identifier: NCT03038399

Published
2022

79. Additional file 1 of Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging

Author

Batra, Abhinandan, Barnard, Alison M., Lott, Donovan J., Willcocks, Rebecca J., Forbes, Sean C., Chakraborty, Saptarshi, Daniels, Michael J., Arbogast, Jannik, Triplett, William, Henricson, Erik K., Dayan, Jonathan G., Schmalfuss, Carsten, Sweeney, Lee, Byrne, Barry J., McDonald, Craig M., Vandenborne, Krista, and Walter, Glenn A.

Subjects
musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities
Abstract

Additional file 1: Global mid ventricular strain (εcc %) in unaffected controls (n=15) and individuals with DMD (n=58) at baseline.

Published
2022
Full Text
View/download PDF

81. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

Author

McDonald, Craig M, primary, Muntoni, Francesco, additional, Penematsa, Vinay, additional, Jiang, Joel, additional, Kristensen, Allan, additional, Bibbiani, Francesco, additional, Goodwin, Elizabeth, additional, Gordish-Dressman, Heather, additional, Morgenroth, Lauren, additional, Werner, Christian, additional, Li, James, additional, Able, Richard, additional, Trifillis, Panayiota, additional, Tulinius, Már, additional, Ryan, M, additional, Jones, K, additional, Goemans, N, additional, Campbell, C, additional, Mah, JK, additional, Selby, K, additional, Chabrol, B, additional, Pereon, Y, additional, Voit, T, additional, Gidaro, T, additional, Schara, U, additional, Kirschner, JB, additional, Nevo, Y, additional, Comi, GP, additional, Bertini, E, additional, Mercuri, E, additional, Colomer, J, additional, Nascimento, A, additional, Vilchez, JJ, additional, Tulinius, M, additional, Sejersen, T, additional, Muntoni, F, additional, Bushby, K, additional, and Guglieri, M, additional

Published
2022
Full Text
View/download PDF

83. Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Author

Shieh, Perry B, primary, Elfring, Gary, additional, Trifillis, Panayiota, additional, Santos, Claudio, additional, Peltz, Stuart W, additional, Parsons, Julie A, additional, Apkon, Susan, additional, Darras, Basil T, additional, Campbell, Craig, additional, McDonald, Craig M, additional, Barohn, Richard J, additional, Bertini, Enrico, additional, Bushby, Kate, additional, Chabrol, Brigitte, additional, Ciafaloni, Emma, additional, Columer, Jaume, additional, Comi, Giacomi Pietro, additional, Connolly, Anne, additional, Finkel, Richard S, additional, Flanigan, Kevin M, additional, Goemans, Nathalie, additional, Guglieri, Michela, additional, Iannaccone, Susan T, additional, Jones, Kristi J, additional, Kaufmann, Petra, additional, Kirschner, Janbernd, additional, Mah, Jean K, additional, Mathews, Katherine, additional, Mercuri, Eugenio, additional, Muntoni, Francesco, additional, Nevo, Yoram, additional, Osorio, Andrés Nascimento, additional, Péréon, Yann, additional, Quinlivan, Rosaline, additional, Renfroe, J. Ben, additional, Russman, Barry, additional, Ryan, Monique, additional, Sampson, Jacinda, additional, Schara, Ulrike, additional, Selby, Kathryn, additional, Sejersen, Thomas, additional, Sproule, Douglas M, additional, Sweeney, H. Lee, additional, Tulinius, Már, additional, Vilchez, Juan J, additional, Vita, Giuseppe, additional, Voit, Thomas, additional, Burns-Wechsler, Stephanie, additional, Wong, Brenda, additional, Abresch, Ted, additional, Henricson, Erik K, additional, Coleman, Kim, additional, Eagle, Michelle, additional, Florence, Julaine, additional, Gappmaier, Ed, additional, McDonald, Craig, additional, Abdel-Hamid, Hoda Z, additional, Bloetzer, Clemens, additional, Butterfield, Russell J, additional, Chae, Jong-Hee, additional, Dastgir, Jahannaz, additional, Desguerre, Isabelle, additional, Escobar, Raul G, additional, Finanger, Erika, additional, Heydemann, Peter, additional, Hughes, Imelda, additional, Kaminska, Anna, additional, Karachunski, Peter, additional, Kudr, Martin, additional, Lotze, Timothy, additional, de Queiroz Campos Araujo, Alexandra Prufer, additional, Dutra de Resende, Maria Bernadete, additional, Tennekoon, Gihan, additional, Topaloglu, Haluk, additional, Torricelli, Ricardo Erazo, additional, Alfano, Lindsay N, additional, James, Meredith K, additional, Lowes, Linda, additional, Mayhew, Anna, additional, Mazzone, Elena S, additional, Nelson, Leslie, additional, and Rose, Kristy J, additional

Published
2021
Full Text
View/download PDF

87. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy

Author

Campbell, Craig, primary, McColl, Elaine, additional, McDermott, Michael P., additional, Martens, William B., additional, Guglieri, Michela, additional, Griggs, Robert C., additional, Straub, Volker, additional, Childs, Anne-Marie, additional, Ciafaloni, Emma, additional, Shieh, Perry B., additional, Spinty, Stefan, additional, Butterfield, Russell J., additional, Horrocks, Iain, additional, Roper, Helen, additional, Maggi, Lorenzo, additional, Baranello, Giovanni, additional, Flanigan, Kevin M., additional, Kuntz, Nancy L., additional, Manzur, Adnan Y., additional, Darras, Basil T., additional, Kang, Peter, additional, Mah, Jean K., additional, Mongini, Tiziana, additional, Ricci, Federica, additional, Morrison, Leslie, additional, Krzesniak-Swinarska, Monika, additional, von der Hagen, Maja, additional, Finkel, Richard S., additional, Kumar, Ashutosh, additional, Wicklund, Matthew, additional, McDonald, Craig M., additional, Henricson, Erik K., additional, Schara-Schmidt, Ulrike, additional, Wilichowski, Ekkehard, additional, Barohn, Richard J., additional, Statland, Jeffrey, additional, Kirschner, Janbernd, additional, Vita, Giuseppe, additional, Vita, Gian Luca, additional, Howard, James F., additional, Hughes, Imelda, additional, McMillan, Hugh J., additional, Pegoraro, Elena, additional, Bello, Luca, additional, Burnette, W. Bryan, additional, Thangarajh, Mathula, additional, and Chang, Taeun, additional

Published
2021
Full Text
View/download PDF

88. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

Author

Landfeldt, Erik, primary, Iff, Joel, additional, Henricson, Erik, additional, Vishwanathan, V., additional, Chidambaranathan, S., additional, Biggar, W. Douglas, additional, McAdam, Laura C., additional, Mah, Jean K., additional, Tulinius, Mar, additional, Cnaan, Avital, additional, Morgenroth, Lauren P., additional, Leshner, Robert, additional, TesiRocha, Carolina, additional, Thangarajh, Mathula, additional, Duong, Tina, additional, Kornberg, Andrew, additional, Ryan, Monique, additional, Nevo, Yoram, additional, Dubrovsky, Alberto, additional, Clemens, Paula R., additional, Abdel-Hamid, Hoda, additional, Connolly, Anne M., additional, Pestronk, Alan, additional, Teasley, Jean, additional, Bertorini, Tulio E., additional, Webster, Richard, additional, Kolski, Hanna, additional, Kuntz, Nancy, additional, Driscoll, Sherilyn, additional, Bodensteiner, John B., additional, Carlo, Jose, additional, Gorni, Ksenija, additional, Lotze, Timothy, additional, Day, John W., additional, Karachunski, Peter, additional, Henricson, Erik K., additional, Abresch, Richard T., additional, Joyce, Nanette C., additional, and McDonald, Craig M., additional

Published
2021
Full Text
View/download PDF

90. OUTCOME RELIABILITY IN NON-AMBULATORY BOYS/MEN WITH DUCHENNE MUSCULAR DYSTROPHY

Author

CONNOLLY, ANNE M., MALKUS, ELIZABETH C., MENDELL, JERRY R., FLANIGAN, KEVIN M., MILLER, PHILIP J., SCHIERBECKER, JEANINE R., SIENER, CATHERINE A., GOLUMBEK, PAUL T., ZAIDMAN, CRAIG M., MCDONALD, CRAIG M., JOHNSON, LINDA, NICORICI, ALINA, KARACHUNSKI, PETER I., DAY, JOHN W., KELECIC, JASON M., LOWES, LINDA P., ALFANO, LINDSAY N., DARRAS, BASIL T., KANG, PETER B., QUIGLEY, JANET, PASTERNAK, AMY E., and FLORENCE, JULAINE M.

Published
2015
Full Text
View/download PDF

92. Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy.

Author

Iff, Joel, Gerrits, Charles, Zhong, Yi, Tuttle, Edward, Birk, Erica, Zheng, Yeya, Paul, Xander, Henricson, Erik K., McDonald, Craig M., and CINRG-DNHS Investigators

Abstract

Introduction/aims: Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States-approved treatment for patients with DMD and exon 51 skip-amenable mutations. Previous analyses have shown that eteplirsen is associated with a statistically significant attenuation of pulmonary decline. In this study we evaluate the effect of eteplirsen treatment from newly available data sources on pulmonary function over time in patients with DMD.Methods: We used a post hoc pooled analysis to compare the percentage of predicted forced vital capacity (FVC%p) and projected time with pulmonary function milestones in patients with DMD and exon 51 skip-amenable mutations receiving eteplirsen (Studies 204 and 301) or standard of care (SoC; Cooperative International Neuromuscular Research Group Duchenne Natural History Study). A mixed model for repeated-measures framework was applied to evaluate the impact of eteplirsen.Results: An average annual rate of FVC%p decline for eteplirsen-treated patients was estimated to be 3.47%, a statistically significant attenuation from the 5.95% rate of decline estimated in SoC patients (P = .0001). Using linear extrapolations of the model-estimated decline in FVC%p, the attenuation in FVC%p decline for eteplirsen-treated patients corresponded to a delay of 5.72 years in time to needing continuous ventilation, 3.31 years in time to needing nighttime ventilation, and 2.11 years in time to needing a cough assist device compared with SoC patients.Discussion: The attenuation of FVC%p decline suggests that eteplirsen-treated patients had statistically significant and clinically meaningful attenuations in pulmonary decline compared with SoC patients. [ABSTRACT FROM AUTHOR]

Published
2022
Full Text
View/download PDF

93. First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

Author

Servais, Laurent, primary, Camino, Eric, additional, Clement, Aude, additional, McDonald, Craig M., additional, Lukawy, Jacek, additional, Lowes, Linda P., additional, Eggenspieler, Damien, additional, Cerreta, Francesca, additional, and Strijbos, Paul, additional

Published
2021
Full Text
View/download PDF

94. Corrigendum to 'Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy' [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502] (Neuromuscular Disorders (2020) 30(6) (492–502), (S0960896620301188), (10.1016/j.nmd.2020.05.002))

Author

Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Mah, Jean K., Campbell, Craig, Guglieri, Michela, Muntoni, Francesco, Takeshima, Yasuhiro, McDonald, Craig M., Kahn, Anna, Atkinson, Karen M., Palmer, Jerry P., Utzschneider, Kristina M., Gebremedhin, Tsige, Kernan-Schloss, Abigail, Kozedub, Alexandra, Montgomery, Brenda K., Morse, Emily J., Mather, Kieren J., Garrett, Tammy, Hannon, Tamara S., Lteif, Amale, Patel, Aniket, Chisholm, Robin, Moore, Karen, Pirics, Vivian, and Pratt, Linda

Abstract

This article reported on the results from a phase 2 trial of domagrozumab and its open-label extension in patients with Duchenne muscular dystrophy (Clinicaltrials.gov identifiers: NCT02310763 and NCT02907619). The manuscript also provided results on two secondary endpoints for magnetic resonance imaging (MRI), muscle volume and muscle volume index. The authors regret that, following publication of the results and in preparation for a separate publication on MRI results from this trial, the MRI images were reviewed and segmentation errors were identified. As a result, the team worked to (1) Perform a rigorous quality inspection of all analysed data; (2) Identify cases where there were incorrect segmentations; (3) correct segmentation errors; (4) Re-analyse all data with correct segmentation. Using the updated MRI data, the MMRM analysis showed there was a change in the significance of secondary endpoints evaluating Thigh Muscle Volume and Muscle Volume Index. No significant differences between treatment groups in muscle volume measures were found in the original analysis. These results have not altered the overall interpretation of the study results but do necessitate revisions to the article. These data confirm that the trial design and execution adequately tested the hypothesis that myostatin inhibition would slow or delay the loss of function in patients with Duchenne muscular dystrophy (DMD). The increase in muscle volume observed by MRI in patients with DMD treated with domagrozumab is in accordance with mechanism of action for domagrozumab, which targets myostatin, a negative regulator of muscle growth. The increase in muscle volume did not lead to a clinical benefit in patients with DMD. The primary endpoint (4 stair climb) did not meet statistical significance, nor did the other functional tests. The study was terminated due to lack of efficacy. Full details of the needed revisions are as follows: 1. In the results section 3.6 (page 8, second paragraph), we reported no significant differences in mean percent change from baseline between domagrozumab and placebo for both muscle volume and muscle volume index. This paragraph was replaced with the following text: “There was a significant difference between domagrozumab and placebo in the mean percent change from baseline in thigh muscle volume at Week 17 (difference 2.945%, P=0.0087) and Week 49 (differences 4.087%, P=0.0298), and in muscle volume index at Week 33 (difference 2.612%, P=0.0376) and Week 49 (differences3.208%, P=0.0411).” 2. In the discussion (page 9), the following sentence, “Although neither muscle volume nor muscle volume index measures were statistically significant in this study, they are both consistent with a potential anabolic effect.” was replaced with, “The increase in muscle volume observed on MRI in patients with DMD treated with domagrozumab, is in accordance with mechanism of action for this compound which targets myostatin, a negative regulator of muscle growth. However, the increase in muscle volume did not lead to a clinical benefit (improved function) in patients with DMD.” 3. In view of the correction to the Results section, this is now reflected in the abstract which has changed to read: “There were no significant between-group differences in secondary clinical endpoints, except for the thigh muscle volume and muscle volume index measures (P

Published
2021

100. Ataluren treatment of patients with nonsense mutation dystrophinopathy

Author

Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P., Connolly, Anne M., Day, John W., Flanigan, Kevin M., Goemans, Nathalie, Jones, Kristi J., Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B., Russman, Barry, Ryan, Monique M., Tulinius, Mar, Voit, Thomas, Moore, Steven A., Lee Sweeney, H., Abresch, Richard T., Coleman, Kim L., Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M., Henricson, Erik, Barth, Jay, Elfring, Gary L., Reha, Allen, Spiegel, Robert J., Oʼdonnell, Michael W., Peltz, Stuart W., and Mcdonald, Craig M.

Published
2014
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results