News

Extracellular RNAs found in urine may be able to serve as non-invasive biomarkers of how patients with muscular dystrophy — especially those with an adult-onset and the more common Duchenne MD form — are responding to therapy, a study reports. Urine-sample tests for these RNAs may also lessen the need…

A pivotal Phase 3 trial of edasalonexent, a potential oral disease-modifying treatment for Duchenne muscular dystrophy (DMD) regardless of mutation type, is enrolling boys ages 4 to 7 in the U.S. and will soon begin with those in Australia, Canada, Europe, and Israel. “We named the trial ‘Polaris’ because it…

Jiffy Lube raised more than $600,000 during its annual Muscle Up! drive, which continued its ongoing support of the Muscular Dystrophy Association (MDA) Summer Camp program, MDA Medical Care Centers, research efforts, and patient help groups. Since its launch in 2012, the campaign has raised $6 million for the…

Expanding its network of institutions that provide standardized, comprehensive care for Duchenne patients, the Parent Project Muscular Dystrophy (PPMD) has named Children’s National Health System its newest Certified Duchenne Care Center (CDCC). This most recent designation brings to 20 the number of clinics to pass muster since the CDCC…

The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work as it should in Duchenne muscular dystrophy and acts to prevent, rather than aid, the regeneration of muscle cells in DMD patients, a study reports. A member of this pathway, a…

Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy for Duchenne muscular dystrophy (DMD), according to preclinical data. The company presented the results in August in two scientific posters at the 2018 Gordon Research Conference on…

A campaign hopes to raise awareness of Duchenne muscular dystrophy (DMD) and promote research toward a cure by asking supporters to paint a nail on one of their little fingers blue. The Pinkies Up campaign is gearing up for November. (Photo courtesy of CureDuchenne) Called “Pinkies…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

Muscle biopsies can benefit the diagnosis and treatment of neuromuscular diseases in children, particularly those with muscular dystrophy or inflammatory muscle disease, a study suggests. In the study, “Does muscle biopsy change the treatment of…

To raise money for treatments and to support families touched by muscular dystrophy, the Muscular Dystrophy Association’s (MDA) New York Muscle Team will present its 22nd annual gala Dec. 3 in New York City. To be held at Cipriana Wall Street, the event will feature former New York…