Sarepta Therapeutics (NASDAQ:SRPT) reported three-year top-line results from its EMBARK Phase 3 pivotal trial evaluating ...

ETHealthworld.com brings latest duchenne muscular dystrophy news, views and updates from all top sources for the Indian Health industry.

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Sarepta Therapeutics SRPT announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating ...

WALTHAM, Mass., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...

Slowing the loss of ambulation in patients with Duchenne muscular dystrophy (DMD) may also mitigate worsening disease burden and overall function, according to a pair of posters presented at ISPOR ...

Keros Therapeutics KROS is a clinical-stage biopharmaceutical company developing innovative therapies for patients with ...

In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...

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 ...

Dyne Therapeutics leverages its FORCE platform to target genetically driven muscle diseases. Read why I am bullish about DYN ...

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...