Fellowship · Sacramento, CA
Sponsored by University of California Davis Health · Directors Cosmo Kwok and Loren Davidson
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Nature medicine · 2025 · 143 citations · Craig McDonald
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
The Lancet. Neurology · 2024 · 113 citations · Craig McDonald
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Neurology · 2024 · 70 citations · Craig McDonald
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA · 2022 · 116 citations · Craig McDonald
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA neurology · 2022 · 114 citations · Craig McDonald
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Annals of neurology · 2023 · 86 citations · Craig McDonald
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
JAMA network open · 2022 · 76 citations · Craig McDonald
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.
Frontiers in cell and developmental biology · 2023 · 71 citations · Craig McDonald