426 results on '"Lowes, Linda"'
Search Results
52. 130 Safety, β-sarcoglycan expression and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.SGCB in LGMD2E/R4
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
62. Longitudinal Effect of Eteplirsen versus Historical Control on Ambulation in Duchenne Muscular Dystrophy
63. Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases
64. Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic
65. Physics-guided Learning for In-home Gait Health Monitoring using Patient-induced Floor Vibrations
66. Validity and Reliability of the Neuromuscular Gross Motor Outcome
67. RELIABILITY AND VALIDITY OF ACTIVE-SEATED: AN OUTCOME IN DYSTROPHINOPATHY
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
70. Reliability and construct validity of the Duchenne Video Assessment
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)
80. Structure- and Sampling-Adaptive Gait Balance Symmetry Estimation Using Footstep-Induced Structural Floor Vibrations
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
82. Spatial, But Not Temporal, Kinematics of Spontaneous Upper Extremity Movements Are Related to Gross and Fine Motor Skill Attainment in Infancy
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
85. CORRELATION OF KNEE STRENGTH TO FUNCTIONAL OUTCOMES IN BECKER MUSCULAR DYSTROPHY
86. KNEE EXTENSOR STRENGTH EXHIBITS POTENTIAL TO PREDICT FUNCTION IN SPORADIC INCLUSION-BODY MYOSITIS
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
99. Activity monitoring in neuromuscular disease: Successes, challenges, and a path forward (P5.6-016)
100. Early signs of gross motor delay in very young boys with Duchenne muscular dystrophy (P1.6-068)
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