Solid Biosciences advances gene therapies for muscular and cardiac diseases, gaining FDA clearance for SGT-212 and targeting ...

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they ...

Study results provide root explanations for how dystrophin functions on a molecular level, providing base-level insights into ...

Deramiocel is a cell therapy that has healing effects in muscle cells. If approved, deramiocel would be a once-quarterly ...

UC Davis Health has premiered a new documentary on a family’s urgent journey across the globe to get their toddler in a ...

Irodanoprost is under clinical development by Mesentech and currently in Phase I for Duchenne Muscular Dystrophy.

New study reveals crucial protein interactions in Duchenne Muscular Dystrophy, opening doors to more precise and effective ...

But if this therapy works, it will heal the muscle." For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure ...

An estimated 1 in 8,000 individuals, or 870,000 people worldwide, are affected by Facioscapulohumeral muscular dystrophy ...

“We are starting very humble with targeting one or two muscles. But if this therapy works, it will heal the muscle.” For nearly 20 years, Spuler and her collaborators have been working to understand ...