Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD ... over with the progressive muscle-wasting disease, which mostly ...

Santhera's Duchenne muscular dystrophy (DMD) therapy Agamree has ... as an alternative to corticosteroids that are used to reduce muscle inflammation and maintain muscle strength and function ...

Marcos Reyes and Valeria Martínez are in a tough spot, unsure which of their twins should receive the treatment they ...

“When I talk about my mum, most people have little or no understanding of what it means to live with a muscle wasting condition,” explains Alex. “Some people I speak to have heard of Duchenne muscular ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).