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A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal...

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NURTURE)

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen admini...

A Study With SAGE-547 for Super-Refractory Status Epilepticus

This is a randomized, double-blind, placebo-controlled trial, designed to evaluate the efficacy and...

Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)

The main objective of this study is to provide confirmatory evidence of efficacy of eteplirsen (AVI-...

Environmental and Genetic Risk Factors for Pediatric Multiple Sclerosis

The purpose of this study is to better understand multiple sclerosis (MS) in children and adolescent...

Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)

The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three w...

Prevention of West Syndrome With Low-dose Adrenocorticotropin Hormone (ACTH)

West syndrome (WS) is a specific type of epilepsy (or seizure disorder) that has three features: i...

Showing 1 - 7 of 7