Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...

Barbie Ferreira and John Leguizamo are broken souls who meet on the Internet in Tracie Laymon's affectingly authentic movie.

A grandmother joined her son and some family friends to abseil 100 feet down from the Falkirk Wheel to raise vital cash for ...

Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship gene therapy, ...

The sky is the limit for Co Antrim pilot Matthew Monaghan who has refused to take no for an answer his entire life after ...

An Indiana man is proving that determination can break down barriers by starting his own company despite life's challenges.

Taking her family’s struggle with a terminal illness and creating a non-profit to help others going through the same ...

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 ...