Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

Absent a federal committee’s recommendation, Duchenne muscular dystrophy advocates are trying to get states to adopt ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the middle ...

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it failed against efficacy targets. Pfizer has been evaluating domagrozumab under the codename PF ...

Ellen J. Annexstad (born 1973) is a paediatrician with expertise in paediatric neurology, and a senior consultant. She is on the PhD programme at the Centre for Congenital Muscular Disorders, where ...

Artificial intelligence (AI)–based electrocardiogram interpretation (AI-ECG) detected left ventricular systolic dysfunction ...

One visit to Virginia Peninsula Community College’s Historic Triangle Campus last summer eased any concerns they might have had. Soon after entering the building, Nicholas and his mother, Liz, were ...

The Buckinghamshire man went to see his local GP. Ryan went from blood tests to muscle biopsies to DNA tests, trying to get ...

Scientists have uncovered how a protein helps build and maintain vital brain connections, providing insights into the ...

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