News

Summit Therapeutics has confirmed plans for an extension stage of a Phase 2 clinical trial evaluating ezutromid as a treatment for boys with Duchenne muscular dystrophy (DMD). …

Data from RECENSUS, a medical chart review of boys with X-linked myotubular myopathy (XLMTM), demonstrates the significant medical burden for children living with the disease, a well as their families and caregivers. Alan Beggs, PhD, director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital, presented data…

Akashi Therapeutics has received an all-clear signal from the U.S. Food and Drug Administration to resume developing a Duchenne muscular dystrophy (DMD) treatment. The FDA had ordered a hold on the development of delayed-release halofuginone (HT-100) after a clinical-trial participant began experiencing life-threatening adverse effects two weeks after beginning the therapy. HT-100 is…

For years, Lexi Pappas hid the fact she had facioscapulohumeral dystrophy (FSHD) — a hereditary muscle wasting disorder that begins in the face and gradually spreads to the shoulders and upper arms, and sometimes also to the legs. As the disease took its toll on Lexi — causing her…

Parent Project Muscular Dystrophy (PPMD) is promoting a series of running events this year to raise money for research to help end Duchenne muscular dystrophy (DMD). The next Run For Our Sons event will be an official charity partner of the Rock n’ Roll Raleigh Marathon, Half…

Researchers have received $119,999 in funding to test the effectiveness of a gene therapy approach in Charcot-Marie-Tooth neuropathy X type 1 (CMTX1), the second most common form of Charcot-Marie-Tooth disease (CMT). The award was granted by the Muscular Dystrophy Association (MDA) and the Charcot-Marie-Tooth Association (CMTA) to Kleopas…

PTC Therapeutics, Inc. has entered into an asset purchase agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort), which was approved in February by the U.S. Food Drug Administration to treat Duchenne muscular dystrophy patients 5 years and older, regardless of their genetic mutation. News about…

A Napa Valley fundraiser in Newport Beach, California, earlier this month attracted more than 400 people and raised at least $1 million for Duchenne muscular dystrophy (DMD) research. The Napa in Newport wine auction on March 4 supported the mission of the nonprofit group CureDuchenne — to improve the lives…

The balance of children with Duchenne muscular dystrophy who are moving becomes worse as the disease progresses, suggesting that balanced-focused interventions may improve the children’s ability to handle everyday tasks. The study, by researchers at Hacettepe University in Turkey, noted that Duchenne progresses differently in different children. That suggests…

Catabasis Pharmaceuticals will present results from Part B of the MoveDMD trial of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD). According to a press release, the presentation, “MoveDMD: Phase 1/2 trial of Edasalonexent, an NF-kB Inhibitor, in 4- to 7-Year-Old Patients with Duchenne Muscular…