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 · 116 citations · Full text

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 · 83 citations · Full text

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

Laurent Servais, John Day, Darryl C. De Vivo et al. · Journal of Neuromuscular Diseases · 2024 · 55 citations · Full text

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 · 35 citations · Full text

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy

Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar et al. · Expert Opinion on Biological Therapy · 2022 · 32 citations · Full text