While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a group of diseases that make muscles weaker and less flexible over time. Duchenne muscular dystrophy (DMD) is the most common type.

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Researchers at the Experimental and Clinical Research Center in Berlin are developing a targeted treatment for muscular dystrophy with the help of gene-editing. Preclinical research led by the Spuler ...

NSAA measures functional motor abilities in children with Duchenne. Also Read: Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development Despite being one year older (average age ...

Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after getting confirmation it will be covered by the NHS in all four nations.