News

Duchenne MD Gene Therapy Project Awarded More Than $2M from Stem Cell Agency

California Stem Cell Agency (CIRM) awarded a total of $2,150,400, to scientists at the University of California Los Angeles (UCLA), Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and the Center for Duchenne Muscular Dystrophy at UCLA, for a cutting-edge stem cell gene therapy research project that could produce a treatment for Duchenne muscular dystrophy (MD).

Duchenne UK Launches ‘World’s Strongest Boys’

Duchenne UK is launching a new video fundraising and awareness campaign for Duchenne muscular dystrophy (DMD) called ‘The World’s Strongest Boys’. The campaign is already supported by British sports celebrities including Jessica Ennis-Hill and Owen Farrell to help boys living with DMD think like heroes by focusing on…

MDA Team Momentum to Beat MD at BMW Dallas Marathon

For the third consecutive year, the Muscular Dystrophy Association‘s nationwide endurance program, the MDA Team Momentum, is revving to run in the Dec. 11, 2016 BMW Dallas Marathon, Half Marathon and Behringer Relay . So far, more than 150 participants on 61 registered teams are geared up already for MDA – even before registrations begins. Since 2014, more…

SIDEROS Clinical Trial Evaluating Raxone for Duchenne MD Is Updated by Santhera

The U.S. FDA has corresponded with Santhera Pharmaceuticals regarding Santhera’s proposed subpart H approval pathway of Raxone (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are not taking concomitant glucocorticoids. Santhera had proposed that results of the SIDEROS clinical trial provide confirmatory evidence of efficacy in these patients while…

Catabasis Launches MoveDMD Open-label Extension Trial for Duchenne MD Therapy

Catabasis Pharmaceuticals has initiated an open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial investigating edasalonexent (CAT-1004) in the treatment of boys with Duchenne muscular dystrophy (DMD). Edasalonexent is a non-corticosteroid, oral small molecule investigational drug that inhibits activated NF-ĸB. In boys with Duchenne MD, the…

Capricor Reaches 50% Enrollment for Duchenne MD HOPE Clinical Trial

Capricor Therapeutics has reached half its target enrollment for its ongoing HOPE-Duchenne clinical trial, the company announced. Capricor discovers, develops, and commercializes first-in-class therapeutics such as the cardiac cell therapy (CAP-1002) for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The company expects to complete enrollment by the end of…