Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...

Columnist Betty Vertin is grateful to have the help of a hired caregiver, but it's also been a difficult adjustment, she says.

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...

Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, unveiled positive results in its sevasemten programme for Becker and Duchenne muscular dystrophies. The company ...

Not too long ago, Editas Medicine was working on Reni-Cel, an investigational treatment for two rare blood disorders. Although the data from clinical trials for this medicine was promising, Editas ...