Carmen Leon Astudillo

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects with Duchenne Muscular Dystrophy (ENDEAVOR)

Swallowing Degradation in Untreated Natural History Patients with Spinal Muscular Atrophy 1: Establishing an International Registry of Bulbar Dysfunction

A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY WHO EXPERIENCED A PLATEAU OR DECLINE IN FUNCTION AFTER GENE THERAPY

A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY

A Phase 3, Multi national, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study

Swallowing Degradation in Untreated Natural History Patients with Spinal Muscular Atrophy Type 1: Establishing an International Registry of Bulbar Dysfunction

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects with Duchenne Muscular Dystrophy (EMBARK)

CTOA