The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) New Phase 1b data further illustrate potential of salanersen in SMA, ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

The MDA's 75th anniversary conference highlights advancements in gene therapy, precision medicine, and patient-centered care for muscular dystrophy. Discussions focus on equitable access to ...

Mutations that cause spinal muscular atrophy (SMA) may lead to abnormalities in the development of cells in the spinal cord, a study found.

The Ironman In You project aims to raise awareness on muscular dystrophy It also aims to raise money for members of the Muscular Dystrophy Association of Singapore As of June 25, this project had ...

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Scotland has become the first UK nation to screen all newborn babies for spinal muscular atrophy. Here is what you need to ...

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...

Biogen offers a financial assistance program for its treatment Spinraza but there’s a catch: patients must sign away their protected health information to the company, reports the Waco Tribune-Herald.

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...