Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Some types of muscular dystrophy cause irregular heartbeats. Gene therapy. Delandistrogene moxeparvovec (Elevidys) is a gene therapy approved to treat Duchenne MD. It's only prescribed to people ...

Lisa is an advocate for raising awareness of Duchenne Muscular Dystrophy: "There needs to be more awareness of DMD. "If more people were aware, maybe they would be less ignorant towards my son's ...

Duchenne muscular dystrophy (DMD) treatment ifetroban led to improvements in measures of heart function in a Phase 2 clinical ...

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

At just 15 years old, Ragheeb has already made a tremendous impact on his community as an ambassador for Capes for Kids, an ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Cumberland Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug has improved the amount of blood pumped by the heart, ...

Evan McMenamin said his brother Travis, "never lets it get him down" despite the "incredible challenges" he faces ...

Entrada Therapeutics plans to initiate ELEVATE-44-201 trial for ENTR-601-44 in Duchenne muscular dystrophy by Q2 2025 ... to provide tangible benefits for people with this life-shortening disease.” ...

The study evaluated ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease – the leading cause of death in DMD patients. It marks a breakthrough for these patients ...