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

By Adriano Marchese Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy.

Second death linked to Sarepta Therapeutics treatment leaves Duchenne muscular dystrophy community reeling, Elevidys use halted in non-ambulatory patients.

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.

Future Market Insights (FMI) has unveiled a compelling new report forecasting significant expansion in the global fuchs endothelial corneal dystrophy market. The report highlights a surge in market ...

In addition to its GeneTAC ® programs, DT-216P2, in development for patients with Friedreich ataxia, and DT-168, for Fuchs endothelial corneal dystrophy, the company is advancing programs in ...

Katherine Mathews, MD, leads the University of Iowa's Wellstone Muscular Dystrophy Specialized Research Center.

Design Therapeutics will present DT-168 program updates for Fuchs endothelial corneal dystrophy at Eyecelerator on May 2, 2025.

In addition to its GeneTAC ® programs, DT-216P2, in development for patients with Friedreich ataxia, and DT-168, for Fuchs endothelial corneal dystrophy, the company is advancing programs in ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscular weakness and mild intellectual disability.