Design Therapeutics Inc. has described transcription modulators reported to be useful for the treatment of myotonic dystrophy type 1 (DM1) and Fuchs’ dystrophy.

Global, Naval Warfare Japan’s government pushes hard to woo Aussies with advanced frigate “We the Japanese government will guarantee that the whole project, and all the contracts will go ...

A second Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said Sunday.

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

Is the association between Duchenne muscular dystrophy and autism spectrum disorder previously identified due to chance?