News

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…

The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening diseases. The investment reflects the nonprofit organization’s plan to double spending on drug development and clinical trials by 2020. Each year,…

A new article reviewing the effect of Duchenne muscular dystrophy (DMD) on heart function has been published in the Journal of Neuromuscular Diseases. The article titled “Cardiac Involvement Classification and Therapeutic Management in Patients with Duchenne Muscular Dystrophy” proposes a classification for the effect of the disease on the heart and outlines the therapeutic management techniques against heart failure.

Bone health varies greatly among patients with facioscapulohumeral muscular dystrophy (FSHD), according to the results of a small group study, and tests for strength and functionality may identify those with higher risks for decreased bone health. The study “Bone Health in Facioscapulohumeral Muscular Dystrophy: A Cross-Sectional Study” was published…