Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...

When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), ...

Duchenne muscular dystrophy is a serious condition that causes progressive muscle weakness. It affects 2,500 boys and men in ...

Researchers at the Experimental and Clinical Research Center in Berlin are developing a targeted treatment for muscular ...

ECRC researchers developed a CRISPR-based gene therapy targeting dysferlin mutations in muscular dystrophy. By editing and ...

Shares of Capricor Therapeutics ($CAPR) were up 8.41% up on Thursday after the rare diseases-focused biotech said it ...

Deramiocel is a cell therapy that has healing effects in muscle cells. If approved, deramiocel would be a once-quarterly ...

There have been few heartwarming storylines in a year full of struggles for the New York Jets. A team that had Super Bowl ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

On November 22, 2024, the U.S. Food and Drug Administration approved Attruby, a near-complete TTR stabilizer, to reduce cardiovascular death ...