Clinical Trials – Pediatric Neurology

Drug Treatment Study for Boys with Duchenne Muscular Dystrophy

A multi-site, randomized, placebo-controlled, double-blind, multiple ascending subcutaneous dose study to evaluate the safety, tolerability and pharmcokinetics of BMS-986089 in ambulatory boys with Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy Treatment Study

Open-label, multiple-dose, efficacy, safety, and tolerability study of Eteplirsen in subjects with Duchenne muscular dystrophy who participated in study 4658-us-201

Expressive Language Sampling as an Outcome Measure

Expressive language sampling as an outcome measure

Extension Study of Ataluren Treatment for Nonsense Mutation Dystrophinopathy

Phase 3 extension study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy

Fragile X Study: Premutation Carriers

Neurological phenotype in FMR1 premutation carriers

Fragile X Syndrome Treatment with MC01CI Study

A six-week, randomized, multicenter, double-blind, parallel, flexed- and fixed-dose study of MC01CI (Metadoxine Extended-Release) 700 mg and 1400 mg compared with placebo in adults and adolescents with fragile X syndrome

Fragile X Syndrome Treatment With NNZ-2566 Compared to Placebo

Study of drug NNZ-2566 in fragile X syndrome compared to placebo

Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study

Phase 3 efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy