The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other ...

Duchenne muscular dystrophy (DMD) is the most common type ... It was the first targeted treatment for this type of mutation and has been shown to help increase the production of dystrophin.

Duchenne muscular dystrophy (DMD) treatment ifetroban led to improvements in measures of heart function in a Phase 2 clinical ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...

FSHD is the second most common form of muscular dystrophy after the Duchenne type, and affects around 1 million people worldwide, with no approved treatments. It tends to cause muscle wasting in ...

SAN DIEGO, Feb. 10, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

The GoFundMe page was set up to raise money to cover the cost of life-saving treatment for the 14-year-old boys, who suffer ...