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Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...

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 ...

Almost a year after buying Kate Therapeutics, Novartis has scooped up another San Diego-based muscle dystrophy biotech in one of the biggest acquisitions of 2025 so far. The Swiss pharma is paying $12 ...

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, ...

"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?' ...

Muscular dystrophies are a group of inherited disorders that cause progressive breakdown of muscle tissue. Two of the most common types are Duchenne muscular dystrophy (DMD) and Becker muscular ...

People with Duchenne muscular dystrophy have a shorter-than-average life expectancy due to changes in muscles that affect breathing, heart activity, and other functions. However, treatment advances ...