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761 results on '"Mendell J"'

1. P281 Quality of life in adults with dysferlinopathy: international clinical outcome study of dysferlinopathy

Author

Hilsden, H., primary, James, M., additional, Dressman, H Gordish, additional, Day, J., additional, Mendell, J., additional, Torron, R Fernandez, additional, Harms, M., additional, Pestronk, A., additional, Vissing, J., additional, Desai, U., additional, Yoshimura, M., additional, Shin, J., additional, Mozaffar, T., additional, Stojkovic, T., additional, Pegoraro, E., additional, Raivas, J Bevilacqu, additional, Olive, M., additional, Paradas, C., additional, Straub, V., additional, and Mayhew, A., additional

Published
2023
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2. P41 EMBARK, a Phase 3 trial evaluating safety and efficacy of delandistrogene moxeparvovec in DMD: study design and baseline characteristics

Author

Muntoni, F., primary, Mercuri, E., additional, Schara-Schmidt, U., additional, Komaki, H., additional, Richardson, J., additional, Singh, T., additional, Guridi, M., additional, Mason, S., additional, Murphy, A., additional, Yu, L., additional, Reid, C., additional, Darton, E., additional, Wandel, C., additional, and Mendell, J., additional

Published
2023
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8. P125 Quantitative MRI in upper limb muscles of patients with dysferlinopathy: 6-months and 12-months longitudinal data from the natural history Jain COS 2 project

Author

Wilson, I., primary, Reyngoudt, H., additional, de Almeida Araujo, E Caldas, additional, Baudin, P., additional, Marty, B., additional, Bolano-Diaz, C., additional, Diaz-Manera, J., additional, Rufibach, L., additional, Hilsden, H., additional, Querin, G., additional, Pegoraro, E., additional, Mendell, J., additional, Stojkovic, T., additional, Straub, V., additional, Blamire, A., additional, and Carlier, P., additional

Published
2023
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9. P84 Assessing infants & toddlers with neuromuscular disorders under 5 years of age using the Neuromuscular Gross Motor Outcome (GRO)

Author

Iammarino, M., primary, Reash, N., additional, Pietruszewski, L., additional, Smith, M., additional, Lammers, J., additional, Waldrop, M., additional, Tsao, C., additional, Chagat, S., additional, Nicolau, S., additional, Flanigan, K., additional, Connolly, A., additional, Mendell, J., additional, Lowes, L., additional, and Alfano, L., additional

Published
2023
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10. P.128 Integrated analyses of data from clinical trials of delandistrogene moxeparvovec in DMD

Author

Zaidman, C., primary, Shieh, P., additional, Proud, C., additional, McDonald, C., additional, Day, J., additional, Mason, S., additional, Guridi, M., additional, Hu, L., additional, Yu, L., additional, Reid, C., additional, Darton, E., additional, Wandel, C., additional, Richardson, J., additional, Malhotra, J., additional, Singh, T., additional, Rodino-Klapac, L., additional, and Mendell, J., additional

Published
2022
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13. P.134a Phase 1/2a trial of delandistrogene moxeparvovec in patients with DMD: 4-year update

Author

Mendell, J., primary, Sahenk, Z., additional, Lehman, K., additional, Lowes, L., additional, Reash, N., additional, Iammarino, M., additional, Alfano, L., additional, Lewis, S., additional, Church, K., additional, Shell, R., additional, Potter, R., additional, Griffin, D., additional, Pozsgai, E., additional, Hogan, M., additional, Hu, L., additional, Mason, S., additional, Darton, E., additional, and Rodino-Klapac, L., additional

Published
2022
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14. FP.34 Clinical outcome study of dysferlinopathy: correlation between MRI fat fraction in lower limbs and clinical outcome assessments over a 3-year period

Author

Smith, F., primary, Reyngoudt, H., additional, Manera, J. Diaz, additional, James, M., additional, Wilson, I., additional, de Almeida Araujo, E. Caldas, additional, Diaz, C. Bolano, additional, Dressman, H. Gordish, additional, Rufibach, L., additional, Mayhew, A., additional, Jones, K., additional, Campana, E. Salort, additional, Walter, M., additional, Stojkovic, T., additional, Yoshimura, M., additional, Pegoraro, E., additional, Mendell, J., additional, Straub, V., additional, Blamire, A., additional, and Carlier, P., additional

Published
2022
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15. P.170 Safety, β-sarcoglycan expression, and functional outcomes from systemic gene transfer of bidridistrogene xeboparvovec in limb-girdle muscular dystrophy type 2E/R4

Author

Rodino-Klapac, L., primary, Pozsgai, E., additional, Lewis, S., additional, Griffin, D., additional, Meadows, A., additional, Lehman, K., additional, Church, K., additional, Reash, N., additional, Iammarino, M., additional, Sabo, B., additional, Alfano, L., additional, Lowes, L., additional, Neuhaus, S., additional, Li, X., additional, and Mendell, J., additional

Published
2022
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16. P.129 One-year data from ENDEAVOR, a phase 1b trial of delandistrogene moxeparvovec in boys with DMD

Author

Zaidman, C., primary, Proud, C., additional, McDonald, C., additional, Mason, S., additional, Guridi, M., additional, Wang, S., additional, Reid, C., additional, Darton, E., additional, Wandel, C., additional, Lewis, S., additional, Malhotra, J., additional, Griffin, D., additional, Potter, R., additional, Rodino-Klapac, L., additional, and Mendell, J., additional

Published
2022
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19. FP.35 Myostatin concentration is unreliable as a biomarker of disease progression in dysferlinopathy

Author

Moore, U., primary, Simon, E. Fernandez, additional, Day, J., additional, Jones, K., additional, Bharucha-Goebel, D., additional, Pestronk, A., additional, Walter, M., additional, Paradas, C., additional, Stojkovic, T., additional, Bravver, E., additional, Pegoraro, E., additional, Mendell, J., additional, Guglieri, M., additional, Straub, V., additional, and Díaz-Manera, J., additional

Published
2022
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21. FP.34 Clinical outcome study of dysferlinopathy: correlation between MRI fat fraction in lower limbs and clinical outcome assessments over a 3-year period

Author

Smith, F, Reyngoudt, H, Manera, J Diaz, James, M, Wilson, I, de Almeida Araujo, E Caldas, Diaz, C Bolano, Dressman, H Gordish, Rufibach, L, Mayhew, A, Jones, K, Campana, E Salort, Walter, M, Stojkovic, T, Yoshimura, M, Pegoraro, E, Mendell, J, Straub, V, Blamire, A, Carlier, P, Smith, F, Reyngoudt, H, Manera, J Diaz, James, M, Wilson, I, de Almeida Araujo, E Caldas, Diaz, C Bolano, Dressman, H Gordish, Rufibach, L, Mayhew, A, Jones, K, Campana, E Salort, Walter, M, Stojkovic, T, Yoshimura, M, Pegoraro, E, Mendell, J, Straub, V, Blamire, A, and Carlier, P

Abstract

The Jain Foundation COS of dysferlinopathy is an international study in genetically confirmed dysferlinopathy patients, with the aim to identify relevant outcome measures to facilitate trial readiness. Due to its wide range of clinical phenotypes and rates of disease progression, an objective marker to quantify disease progression would be ideal. We assessed the application of quantitative magnetic resonance imaging (MRI) as a prognostic tool for these patients. Our aim is to establish whether there is a correlation between fat fraction (FF) in thigh and/or lower leg muscles and clinical outcome assessments (COA) when comparing baseline (BL) values and changes from BL to year 1 (Y1) and to year 3 (Y3). We selected 84 patients from COS1 who had a Dixon MRI of the lower limbs (LL) and at least one of the following COA: time to rise from floor (RFF), time to climb / descend 4 steps (4SC/4SD), time up and go (TUG), time to walk 10m (10MWT), 6 min walk test (6MWT) and North Star Assessment for limb girdle type muscular dystrophy (NSAD) score. Spearman correlation (rs) was performed using SPSS statistics, p value 0.05. We found a significant correlation at BL between LL FF values and all COA, with the highest rs between thigh FF and NSAD (-.675) and 6MWT (-.665). We didn't find any correlations between changes in FF between BL and Y1 and changes in COA during that same period, but we did observe a significant correlation with changes in TUG (.445) and 4SC (.41) between BL and Y3. We observed a significant correlation between changes in thigh FF between BL and Y3 and changes in TUG (.706), RFF (.607), 4SC (.545) and NSAD[HR1] (-.374). No correlations were found when analysing changes in lower legs FF and COA. Our results show that changes in FF of the thigh muscles over one year could predict functional changes at a later stage, in a three-year period, suggesting that MRI could be used to identify dysferlinopathy patients at risk of more severe disease progression in routi

Published
2022

22. Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach

Author

Mayhew, A. G., James, M. K., Moore, U., Sutherland, H., Jacobs, M., Feng, J., Lowes, L. P., Alfano, L. N., Muni Lofra, R., Rufibach, L. E., Rose, K., Duong, T., Bello, L., Pedrosa-Hernandez, I., Holsten, S., Sakamoto, C., Canal, A., Sanchez-Aguilera Praxedes, N., Thiele, S., Siener, C., Vandevelde, B., Dewolf, B., Maron, E., Gordish-Dressman, H., Hilsden, H., Guglieri, M., Hogrel, J. -Y., Blamire, A. M., Carlier, P. G., Spuler, S., Day, J. W., Jones, K. J., Bharucha-Goebel, D. X., Salort-Campana, E., Pestronk, A., Walter, M. C., Paradas, C., Stojkovic, T., Mori-Yoshimura, M., Bravver, E., Diaz-Manera, J., Pegoraro, E., Mendell, J. R., Jain COS Consortium, Straub, V., Jain Foundation, John Walton Centre Muscular Dystrophy Research Centre, and MRC Centre Neuromuscular Biobank (UK)

Subjects
PROMs, Neurology, quality of life, Neurology (clinical), clinical outcome assessments, dysferlinopathy, limb girdle muscular dystrophy, Function and Dysfunction of the Nervous System
Abstract

Dysferlinopathy is a muscular dystrophy with a highly variable functional disease progression in which the relationship of function to some patient reported outcome measures (PROMs) has not been previously reported. This analysis aims to identify the suitability of PROMs and their association with motor performance.Two-hundred and four patients with dysferlinopathy were identified in the Jain Foundation's Clinical Outcome Study in Dysferlinopathy from 14 sites in 8 countries. All patients completed the following PROMs: Individualized Neuromuscular Quality of Life Questionnaire (INQoL), International Physical Activity Questionnaire (IPAQ), and activity limitations for patients with upper and/or lower limb impairments (ACTIVLIMs). In addition, nonambulant patients completed the Egen Klassifikation Scale (EK). Assessments were conducted annually at baseline, years 1, 2, 3, and 4. Data were also collected on the North Star Assessment for Limb Girdle Type Muscular Dystrophies (NSAD) and Performance of Upper Limb (PUL) at these time points from year 2. Data were analyzed using descriptive statistics and Rasch analysis was conducted on ACTIVLIM, EK, INQoL. For associations, graphs (NSAD with ACTIVLIM, IPAQ and INQoL and EK with PUL) were generated from generalized estimating equations (GEE). The ACTIVLIM appeared robust psychometrically and was strongly associated with the NSAD total score (Pseudo R 2 0.68). The INQoL performed less well and was poorly associated with the NSAD total score (Pseudo R 2 0.18). EK scores were strongly associated with PUL (Pseudo R 2 0.69). IPAQ was poorly associated with NSAD scores (Pseudo R 2 0.09). This study showed that several of the chosen PROMs demonstrated change over time and a good association with functional outcomes. An alternative quality of life measure and method of collecting data on physical activity may need to be selected for assessing dysferlinopathy., The estimated US $4 million needed to fund this study was provided by the Jain Foundation (www.jainfoundation.org). The John Walton Muscular Dystrophy Research Centre is part of the MRC Centre for Neuromuscular Diseases (Grant Number MR/K000608/1).

Published
2022

25. 425P Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD): a phase 1/2a study.

Author

Mendell, J., Sahenk, Z., Lowes, L., Reash, N., Iammarino, M., Alfano, L., Signorovitch, J., Jin, J., Gao, P., Mason, S., Elkins, J., and Rodino-Klapac, L.

Subjects
*DUCHENNE muscular dystrophy, *NATURAL history, *CHILD patients, *LIVER enzymes, *GENETIC transformation
Abstract

Delandistrogene moxeparvovec, an rAAVrh74-based gene transfer therapy designed to address absent functional dystrophin by delivering a transgene encoding an engineered micro-dystrophin, is approved (US, UAE, Qatar, Kuwait, Bahrain and Oman; Mar 2024) for ambulatory pediatric patients (pts) aged 4 through 5 years (yrs) with Duchenne muscular dystrophy (DMD) with a confirmed DMD mutation. We report 5-yr Study 101 (NCT03375164) results. Four ambulatory pts with DMD (≥4–<8 yrs old) received delandistrogene moxeparvovec (1.33×10¹⁴ vg/kg) and prednisone (1 mg/kg; 30-day taper post-infusion) 1-day pre-infusion. Primary outcome was safety. Change from baseline in North Star Ambulatory Assessment (NSAA) was an efficacy outcome. Collaborative Trajectory Analysis Project (cTAP) model predicted the NSAA natural history trajectory for a group of untreated pts with matching baseline prognostic factors (including age and motor function) as those in Study 101. At 5 yrs post-infusion, mean (range) age was 10.14 (9.02–11.02) yrs; all pts remained ambulant. Overall, 75 adverse events (AEs) were reported (most occurred ≤70 days post-infusion); 18 treatment-related treatment-emergent AEs were reported (all mild or moderate) and the most common were vomiting and increased liver enzymes (all resolved). There were no serious AEs, complement-mediated AEs, study discontinuations, or deaths. At 5 yrs, mean (standard deviation) change in NSAA total score from baseline was +7.5 (2.4) points with a least squares mean difference vs a propensity-score-weighted external control at 4.5 yrs of 8.2 points (P=0.0241). cTAP modeling showed increasing divergence of treated pts' NSAA trajectories vs their natural history predictions over the 5 yrs of follow-up. No new safety signals were reported 5 yrs post-infusion, supporting the manageable safety profile reported previously. NSAA outcomes indicated long-term stabilization of motor function at ages where decline is expected as seen in natural history. [ABSTRACT FROM AUTHOR]

Published
2024
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26. 423P Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy: phase 3 EMBARK primary results.

Author

Mendell, J., Muntoni, F., McDonald, C., Mercuri, E., Ciafaloni, E., Komaki, H., Leon-Astudillo, C., Nascimento, A., Proud, C., Schara-Schmidt, U., Veerapandiyan, A., Zaidman, C., Murphy, A., Reid, C., Asher, D., Darton, E., Mason, S., Fontoura, P., Elkins, J., and Rodino-Klapac, L.

Subjects
*DUCHENNE muscular dystrophy, *ADVERSE health care events, *ANTIBODY titer, *CREATINE kinase, *CHILD patients
Abstract

Delandistrogene moxeparvovec, an rAAVrh74-based gene transfer therapy designed to address absent functional dystrophin by delivering a transgene encoding engineered micro-dystrophin, is approved (US, UAE, Qatar, Kuwait, Bahrain, Oman; Mar 2024) for ambulatory pediatric patients (pts) aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed DMD mutation. We report Part 1 (Week [Wk] 52) results of the two-part EMBARK trial (NCT05096221). Key inclusion criteria: Ambulatory males aged ≥4 <8 years with a confirmed DMD mutation within exons 18–79 (inclusive); screening North Star Ambulatory Assessment (NSAA) score >16 <29; rAAVrh74 antibody titers <1:400; stable daily oral corticosteroid dose (≥12 wks pre-screening). Pts received single-dose delandistrogene moxeparvovec (1.33×10¹⁴ vg/kg, intravenous) or placebo. Primary endpoint: change from baseline to Wk 52 in NSAA total score. At Wk 52 (N=125), the primary endpoint was not statistically significant—least-squares mean between-group difference for delandistrogene moxeparvovec (n=63) vs placebo (n=61) was 0.65 points (P=0.2441). Time to rise, 10-meter walk/run, stride velocity 95th centile and time to ascend 4 steps favored the treatment group (P<0.05, nominal). A prespecified global statistical test on a composite of functional endpoints supported treatment benefit (P=0.0044). Micro-dystrophin was expressed in the treatment group at Wk 12 (western blot). Mean creatine kinase levels decreased from baseline with delandistrogene moxeparvovec vs placebo (P=0.0002, nominal). Treatment-related treatment-emergent adverse events (AEs) included vomiting (54.0%), nausea (31.7%) and decreased appetite (27.0%), with no study discontinuations, deaths or complement-mediated AEs. Based on the totality of functional assessments including timed function tests, treatment with delandistrogene moxeparvovec indicates beneficial modification of disease trajectory. Safety was manageable and consistent with prior experience. [ABSTRACT FROM AUTHOR]

Published
2024
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27. Safety, β-Sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4

Author

Rodino-Klapac, L. R., additional, Pozsgai, E. R., additional, Lewis, S., additional, Griffin, D. A., additional, Meadows, A. S., additional, Lehman, K. J., additional, Church, K., additional, Reash, N. F., additional, Iammarino, M. A., additional, Lowes, L. P., additional, Koenig, E., additional, Neuhaus, S., additional, Li, X., additional, Mueller-York, A., additional, and Mendell, J. R., additional

Published
2021
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28. SMA - TREATMENT

Author

Strauss, K., primary, Muntoni, F., additional, Farrar, M., additional, Saito, K., additional, Mendell, J., additional, Servais, L., additional, McMillan, H., additional, Swoboda, K., additional, Kwon, J., additional, Zaidman, C., additional, Chiriboga, C., additional, Iannaccone, S., additional, Krueger, J., additional, Parsons, J., additional, Shieh, P., additional, Kavanagh, S., additional, Chand, D., additional, Tauscher-Wisniewski, S., additional, and Macek, T., additional

Published
2021
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29. LGMD

Author

Rodino-Klapac, L., primary, Pozsgai, E., additional, Lewis, S., additional, Griffin, D., additional, Meadows, A., additional, Lehman, K., additional, Church, K., additional, Reash, N., additional, Iammarino, M., additional, Lowes, L., additional, Koenig, E., additional, Neuhaus, S., additional, Li, X., additional, and Mendell, J., additional

Published
2021
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30. SMA - TREATMENT

Author

Mendell, J., primary, Finkel, R., additional, Mercuri, E., additional, Strauss, K., additional, Day, J., additional, Kleyn, A., additional, Chand, D., additional, Tauscher-Wisniewski, S., additional, and Meriggioli, M., additional

Published
2021
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31. CLINICAL TRIAL HIGHLIGHTS

Author

Mendell, J., primary, Shieh, P., additional, Sahenk, Z., additional, Lehman, K., additional, Lowes, L., additional, Reash, N., additional, Iammarino, M., additional, Alfano, L., additional, Powers, B., additional, Woods, J., additional, Skura, C., additional, Mao, H., additional, Staudt, L., additional, Potter, R., additional, Griffin, D., additional, Lewis, S., additional, Hu, L., additional, Upadhyay, S., additional, Singh, T., additional, and Rodino-Klapac, L., additional

Published
2021
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32. DMD - TREATMENT

Author

Zaidman, C., primary, Proud, C., additional, McDonald, C., additional, Giblin, K., additional, Collins, L., additional, Wang, S., additional, Upadhyay, S., additional, Lewis, S., additional, Malhotra, J., additional, Griffin, D., additional, Potter, R., additional, Guridi, M., additional, Rodino-Klapac, L., additional, and Mendell, J., additional

Published
2021
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33. LGMD

Author

Moore, U., primary, James, M., additional, Spuler, S., additional, Day, J., additional, Jones, K., additional, Bharucha-Goebel, D., additional, Salort-Campana, E., additional, Pestronk, A., additional, Walter, M., additional, Paradas, C., additional, Stojkovic, T., additional, Yoshimura, M. Mori, additional, Bravver, E., additional, Pegoraro, E., additional, Mendell, J., additional, Bushby, K., additional, Straub, V., additional, and Mayhew, A., additional

Published
2021
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34. DMD - TREATMENT

Author

Mendell, J., primary, Sahenk, Z., additional, Lehman, K., additional, Nease, C., additional, Lowes, L., additional, Reash, N., additional, Iammarino, M., additional, Alfano, L., additional, Vaiea, J., additional, Lewis, S., additional, Church, K., additional, Shell, R., additional, Potter, R., additional, Griffin, D., additional, Pozsgai, E., additional, Hogan, M., additional, and Rodino-Klapac, L., additional

Published
2021
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35. OUTCOME MEASURES

Author

Powers, B., primary, Reash, N., additional, Iammarino, M., additional, Shannon, K., additional, Waldrop, M., additional, Connolly, A., additional, Flanigan, K., additional, Tsao, C., additional, Mendell, J., additional, Lowes, L., additional, and Alfano, L., additional

Published
2021
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37. MicroRNA miR-155 ist ein Biomaker früher Formen des Pankreaskarzinoms

Author

Habbe, N., Koorstra, J. -B. M., Mendell, J. T., Feldmann, G., Mullendore, M. E., Goggins, M. G., Maitra, A., Buhr, H. J., editor, Hölscher, A., editor, Izbicki, J. R., editor, Jauch, K. W., editor, Post, S., editor, Allgayer, H., editor, Bartsch, D. K., editor, Fries, H., editor, Ghadimi, B. M., editor, Habermann, J. K., editor, Kalthoff, H., editor, Schackert, H. K., editor, Bruns, C. J., editor, Duda, D. N., editor, Fändrich, F., editor, Heberer, M., editor, Nüssler, A. K., editor, Stark, G. B., editor, Brückner, U. B., editor, Ertel, W., editor, Faist, E., editor, Heidecke, C. D., editor, Kalff, J. C., editor, Menger, M. D., editor, Schade, U. F., editor, Vollmar, B., editor, Bechstein, W. O., editor, Klar, E., editor, Klempnauer, J., editor, Minor, T., editor, Neuhaus, P., editor, Schmidt, J., editor, Langer, S., editor, Laschke, M. W., editor, Machens, H. G., editor, Steinau, H. U., editor, Südkamp, N., editor, Vogt, P., editor, Berger, D., editor, Klinge, U., editor, Schachtrupp, A., editor, Schilling, M. K., editor, Schumpelick, V., editor, Altendorf-Hofmann, A., editor, Bollschweiler, E., editor, Bruch, H. -P., editor, Lehnert, T., editor, Lorenz, W., editor, Neugebauer, E., editor, Ohmann, C., editor, Seiler, C. M., editor, Becker, H., editor, Betzler, M., editor, Bittner, R., editor, Broelsch, C., editor, Büchler, M. W., editor, Bumm, R., editor, Dralle, H., editor, Friess, H., editor, Frilling, A., editor, Germer, C. T., editor, Hopt, U., editor, Jonas, S., editor, Jost, J., editor, Markus, B., editor, Raab, H. R., editor, Reith, H. B., editor, Schlag, P. M., editor, Teichmann, W., editor, Uhl, W., editor, Wenisch, H., editor, and Zornig, C., editor

Published
2009
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43. OUTCOME MEASURES

Author

Iammarino, M., primary, Miller, N., additional, Alfano, L., additional, Powers, B., additional, Shannon, K., additional, Connolly, A., additional, Waldrop, M., additional, Tsao, C., additional, Sahenk, Z., additional, Flanigan, K., additional, Mendell, J., additional, and Lowes, L., additional

Published
2020
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44. SMA – THERAPY

Author

Chand, D., primary, Finkel, R., additional, Day, J., additional, Darris, B., additional, Kuntz, N., additional, Connolly, A., additional, Zaidman, C., additional, Crawford, T., additional, Butterfield, R., additional, Shieh, P., additional, Tennekoon, G., additional, Brandesma, J., additional, Iannaccone, S., additional, Meriggioli, M., additional, Tauscher-Wisniewski, S., additional, Shoffner, J., additional, Ogrinc, F., additional, Kavanagh, S., additional, Feltner, D., additional, and Mendell, J., additional

Published
2020
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45. DMD – THERAPY

Author

Mendell, J., primary, Sahenk, Z., additional, Lehman, K., additional, Nease, C., additional, Lowes, L., additional, Miller, N., additional, Iammarino, M., additional, Alfano, L., additional, Vaiea, J., additional, Al-Zaidy, S., additional, Lewis, S., additional, Church, K., additional, Shell, R., additional, Potter, R., additional, Griffin, D., additional, Pozsgai, E., additional, Hogan, M., additional, and Rodino-Klapac, L., additional

Published
2020
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47. FROM THE SPINAL CORD TO THE MUSCLE

Author

Moore, U., primary, Gordish, H., additional, Maneraz, J. Díaz, additional, James, M., additional, Mayhew, A., additional, Guglieri, M., additional, Spuler, S., additional, Day, J., additional, Jones, K., additional, Bharucha-Goebel, D., additional, Salort-Campana, E., additional, Pestronk, A., additional, Walter, M., additional, Paradas, C., additional, Stojkovic, T., additional, Yoshimura, M., additional, Bravver, E., additional, Pegoraro, E., additional, Mendell, J., additional, and Straub, V., additional

Published
2020
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48. LIMB GIRDLE MUSCULAR DYSTROPHIES

Author

Rodino-Klapac, L., primary, Pozsgai, E., additional, Lewis, S., additional, Griffin, D., additional, Meadows, A., additional, Lehman, K., additional, Church, K., additional, Miller, N., additional, Iammarino, M., additional, Lowes, L., additional, and Mendell, J., additional

Published
2020
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49. LIMB GIRDLE MUSCULAR DYSTROPHIES

Author

Miller, N., primary, Iammarino, M., additional, Alfano, L., additional, Powers, B., additional, Shannon, K., additional, Nease, C., additional, Lehman, K., additional, Mendell, J., additional, and Lowes, L., additional

Published
2020
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50. SMA – THERAPY

Author

Mendell, J., primary, Shell, R., additional, Lehman, K., additional, McColly, M., additional, Lowes, L., additional, Alfano, L., additional, Miller, N., additional, Iammarino, M., additional, Church, K., additional, Ogrinc, F., additional, Ouyang, H., additional, Kernbauer, E., additional, Joshi, S., additional, Sproule, D., additional, Meriggioli, M., additional, Feltner, D., additional, and Al-Zaidy, S., additional

Published
2020
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