Crystal M. Proud

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et al. · Nature Medicine · 2024 · 157 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +15

Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to &lt;8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study <scp>SRP</scp>‐9001‐103 (<scp>ENDEAVOR</scp>)

Craig M. Zaidman, Crystal M. Proud, Craig M. McDonald et al. · Annals of Neurology · 2023 · 91 citations · Full text

Medicine, Molecular Medicine, Translational Sciences +10

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Laurent Servais, Darryl C. De Vivo, Janbernd Kirschner et al. · Journal of Neuromuscular Diseases · 2024 · 65 citations · Full text

Medicine, Molecular Medicine, Pharmacotherapy +14

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment

Olga Mitelman, Hoda Abdel‐Hamid, Barry J. Byrne et al. · Journal of Neuromuscular Diseases · 2021 · 52 citations · Full text

Medicine, Pharmacotherapy, Rehabilitation Medicine +16

Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy

Jerry R. Mendell, Crystal M. Proud, Craig M. Zaidman et al. · Pediatric Neurology · 2024 · 41 citations · Full text

Medicine, Biotechnology, Molecular Biology +16