Maitea Guridi

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

Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials

Laurent Servais, Karl Yen, Maitea Guridi et al. · Journal of Neuromuscular Diseases · 2021 · 79 citations · Full text

Medicine, Rehabilitation Medicine, Functional Movement Assessment +15

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy

Francesco Muntoni, Barry J. Byrne, Hugh J. McMillan et al. · Neurology and Therapy · 2024 · 23 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +14

Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

Hannah Staunton, Claire Trennery, Rob Arbuckle et al. · Health and Quality of Life Outcomes · 2021 · 19 citations · Full text

Medicine, Neurology, Evidence-based Practice +14