In a major milestone for Capricor Therapeutics, the company has announced that the U.S. Food and Drug Administration (FDA) ...

Dyne Therapeutics (NasdaqGS:DYN) has initiated the Phase 3 Harmonia trial of DYNE-101 in myotonic dystrophy type 1, following alignment with the FDA on the study design. The Harmonia trial is a ...

New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...

Brooklyn Nets radio play-by-play voice Chris Carrino Sr. was honored by the Naismith Basketball Hall of Fame with a Curt ...

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 ...

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 ...

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 ...

"While we did see correlations between activity levels and cardiomyopathy progression in patients with Duchenne muscular dystrophy, these correlations were not particularly strong. Our interpretation ...

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 ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted ...

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

Nila Morton, a graduate student at Howard University, was excited to be traveling back to South Carolina for Thanksgiving after almost a year of being away from home. Morton, 25, was born with a rare ...