"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

The Muscular Dystrophy Association (MDA) concluded its 2026 MDA Clinical & Scientific Conference yesterday, convening over 2,400 attendees from 40 countries to help shape the future of neuromuscular ...

"I don’t worry about him getting scared away because of my disability," Tina Fegley tells PEOPLE of boyfriend Tyler Waldman Tina Fegley Couple Tina Fegley and Tyler Waldman met on Dateability, a ...

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

Duchenne muscular dystrophy (DMD) is a disorder that causes progressive muscle weakness and a loss of function. The symptoms appear early in life, and most affected individuals require the use of a ...

Omaha Fire Department's annual steak fry aims to raise funds for families affected by muscular disease through their Fill the Boot program.

Delpacibart braxlosiran (AOC 1020), the first investigational therapy to target the underlying cause of FSHD, provided greater than 50% mean reduction across multiple DUX4 regulated gene panels (2 ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Biomarker cohort for delpacibart braxlosiran (del-brax), the first potential therapy to target DUX4, is measuring changes in DUX4 regulated biomarkers; del-brax 2 mg/kg will be administered every six ...