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A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

purpose: This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below: • Group 1, subjects aged <6 years, 100 subjects; • Group 2, subjects aged >=6 years and <12 years, 180 subjects; • Group 3, subjects aged >=12 years, 50 subjects...

sponsor: Pfizer

location: China

A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

purpose:

This study will be comprised of 2 parts: Part A (Multiple Ascending Dose (MAD)) which will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD); Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD in patients who have completed Part A.

sponsor: Sarepta Therapeutics

location: United States

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

purpose:

The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids

sponsor: Santhera Pharmaceuticals

location: United States, Austria, Belgium, Bulgaria, France, Germany, Hungary, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

sponsor: Sarepta Therapeutics

location: United States

A Registered Cohort Study on Duchenne Muscular Dystrophy

purpose:

Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.

sponsor: Ning Wang, MD, PhD

location: China

A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy

purpose: Current DMD therapies are aimed at increasing dystrophin levels and reducing inflammation. Improved anti-inflammatory therapies are needed to safely treat this pathology and delay disease progression. This study will be conducted in a single-centre and assess the safety, efficacy and PK of ATL1102 in non-ambulatory patients with DMD. Read more:...

sponsor: Antisense Therapeutics Limited

location: Australia

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