Learn About Dystrophinopathy

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However, there may be experts who have treated this or similar conditions in our Find a Doctor section and research may be available in our Latest Advances section.

Who are the top Dystrophinopathy Local Doctors?
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What are the Latest Advances for Dystrophinopathy?
Antioxidants to prevent respiratory decline in people with Duchenne muscular dystrophy and progressive respiratory decline.
Antioxidants to prevent respiratory decline in people with Duchenne muscular dystrophy and progressive respiratory decline.
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What are the latest Dystrophinopathy Clinical Trials?
An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Summary: The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under t...

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A Registered Cohort Study on Duchenne Muscular Dystrophy

Summary: 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 op...