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For more than two decades, researchers at the University of Basel, Switzerland, have been investigating a severe form of muscular dystrophy in which muscles progressively degenerate. The research team ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock is trading higher on Wednesday, with apparently no news to justify the movement. This week, Sarepta will present data from its gene therapy, ...

Partnership leverages AI-powered genome design to optimize AAV constructs, accelerate lead candidate selection, and improve drug substance manufacturability for rare and ultra-rare neuromuscular ...

"I don't like the attention at all," said Shep, as his family and friends call him, who gets a lot of it at school. "It just annoys me, that every day I'm asked, 'Hey, why do you need a wheelchair?' ...

Abstract: Rehabilitation for patients with muscle dystrophy is based on performing a lifelong program of regular exercise in coordination with the therapist. However, traditional physiotherapy can be ...

FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA resubmission Company preparing to resubmit CRL response ...