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An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

—The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra Bonner of the Center for Pediatric Neurosciences at Cleveland Clinic.

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.

Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy treatment. The patient was a young man who suffered acute liver failure ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care needs and physical activity barriers for individuals with myotonic dystrophy.

Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function. Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular ...

Duchenne muscular dystrophy causes progressive muscle degeneration and weakness, but physical exercise should still be part of a comprehensive treatment strategy. Duchenne muscular dystrophy (DMD ...