Vanessa Pataia,  —

At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive results in four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 study. The therapy improved the boys’ motor abilities and…

PerkinElmer Genomics has launched a new test that is able to accurately detect the shortening of the D4Z4 gene region which causes facioscapulohumeral muscular dystrophy (FSHD) type 1.  FSHD affects approximately one in 10,000 to one in…

Researchers have improved a gene editing technique that may more efficiently correct DMD gene mutations in people with Duchenne muscular dystrophy (DMD), restore the normal function of the dystrophin protein, and improve muscle function, a study in…

The Muscular Dystrophy Association (MDA) has launched its 38th annual #MDAShamrocks campaign — the largest St. Patrick’s fundraiser in the U.S. — aiming to raise money and awareness about muscular dystrophy and…

Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open. During the conference, which will be held March 8–10 in Washington, D.C., participants will receive extensive training and support to empower and prepare them to advocate for Duchenne muscular dystrophy…

Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an early Phase 2 clinical trial suggests. The findings were detailed in a study, “…

  Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the world. The Vivli platform, which currently hosts information from more than 4,700 clinical…