News

Duchenne UK announced that it has awarded a sizable grant to Evox Therapeutics to advance in early testing a way of using gene therapy to deliver — without a virus as a transport agent — the dystrophin gene or shorter versions of it to people with Duchenne muscular dystrophy. The group’s…

This month’s Democratic takeover of the U.S. House of Representatives following the Nov. 6 midterm elections could be good news for Americans with rare diseases, says the National Organization for Rare Disorders (NORD), which represents 280 patient advocacy organizations. “The entire community advocated against the repeal of the Affordable Care…

Catabasis Pharmaceuticals will collaborate with the University of Texas Southwestern to investigate potential benefits of edasalonexent on heart function in Duchenne and Becker muscular dystrophies. After the promising results of Catabasis’ Phase 1/2 MoveDMD trial (NCT02439216) and its open-label extension, edasalonexent is now being…

CureDuchenne and Avidity Biosciences will host a webinar Friday to brief the Duchenne muscular dystrophy (DMD) community on Avidity’s preclinical development of DMD therapy candidates. The webinar will take place at 4 p.m. EST and will feature Debra Miller, CureDuchenne’s founder and CEO, and Arthur A. Levin,…

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…