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53. Safety, β-Sarcoglycan Expression, and Functional Outcomes From Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4 (S23.005)

54. Development of Validated Clinical Outcome Assessments in Limb Girdle Muscular Dystrophy R1/2A (P14-13.004)

55. A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of SRP-9001 for Treating Patients with Duchenne Muscular Dystrophy (S23.002)

56. Phase 1/2a Trial of SRP-9001 in Patients with Duchenne Muscular Dystrophy: 3-Year Safety and Functional Outcomes (S23.004)

57. Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy

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

59. eP201: A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy

60. eP212: Safety, β-Sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4

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

63. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases

66. Validity and Reliability of the Neuromuscular Gross Motor Outcome

68. 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

69. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

71. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

72. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

73. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

74. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease

75. Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

76. Comparison of strength testing modalities in dysferlinopathy.

77. INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.

78. Safety, β-sarcoglycan Expression and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in Patients with Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E) (4330)

79. A Multicenter Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy (4478)

81. Use of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance

83. 266th ENMC International Workshop: Remote delivery of clinical care and validation of remote clinical outcome assessments in neuromuscular disorders: A response to COVID-19 and proactive planning for the future. Hoofddorp, The Netherlands, 1–3 April 2022

84. Eteplirsen for the treatment of Duchenne muscular dystrophy

87. Contributors

88. MD-Vibe

89. Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

90. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

91. Natural History of Type 2 and 3 Spinal Muscular Atrophy (SMA): Longitudinal 2-year NatHis-SMA Study (530)

92. Gene-Replacement Therapy in Spinal Muscular Atrophy Type 1: Long-Term Follow-Up From the Onasemnogene Abeparvovec-xioi Phase 1/2a Clinical Trial (1808)

93. Assessment of disease progression in dysferlinopathy. A 1-year cohort study

94. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

95. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy

96. Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial

97. Systemic Gene Transfer with rAAVrh74.MHCK7.SGCB Increased -sarcoglycan Expression in Patients with Limb Girdle Muscular Dystrophy Type 2E

98. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

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