The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD). The review, “Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value,” focuses on Sarepta Therapeutics’ Exondys 51 (eteplirsen) and…
Sarepta Therapeutics is partnering with Roche to accelerate the development and expand the availability of SRP-9001 — its leading gene therapy candidate for Duchenne muscular dystrophy (DMD) — outside the Unites States. DMD is caused by deficient production of dystrophin — an essential protein for…
A Phase 2b trial of the investigational therapy vamorolone is 4-to-6-year-old boys with Duchenne muscular dystrophy (DMD) has begun to enroll patients. Patient recruitment for the international ReveraGen BioPharma-sponsored, double-blind VISION-DMD trial (NCT03439670) is now ongoing. More information on study locations and contacts can be found…
Treatment with investigational cell therapy CAP-1002 improved heart and skeletal muscle function, and reduced cardiac scarring in boys and young men with advanced Duchenne muscular dystrophy (DMD) in a Phase 1/2 trial. Findings also revealed no serious safety issues related to treatment with Capricor Therapeutics’ lead therapy candidate. The…
If it weren’t for a serious auto accident during his senior year of high school, Christopher Anselmo might have had to wait many more years before discovering he had limb-girdle muscular dystrophy (LGMD). This rare condition — a general term for more than 30 muscle-wasting conditions — affects anywhere from…
Parent Project Muscular Dystrophy (PPMD) has published a report outlining key results of its decade-old study of some 4,000 registered Duchenne and Becker muscular dystrophy patients. Representing more than 100 countries, the nonprofit organization’s Ten Year Registry Report is a compilation of data provided by…
To improve patient engagement and care management tools for the Duchenne Registry, Parent Project Muscular Dystrophy (PPMD) has teamed up with THREAD, a company specializing in remote and virtual patient research. Enhancements to the registry — available on both IOS and Android devices — will be mainly…
The U.S. Food and Drug Administration (FDA) selected Wave Life Sciences‘ Phase 2/3 trial assessing the efficacy and safety of WVE-210201 (suvodirsen) in Duchenne muscular dystrophy for its pilot program for complex innovative trial designs. This initiative aims to modernize the design of clinical trials and advance drug development. Wave will…
Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Aldevron, which produces biological products for the biotech industry, will be responsible for supplying…
Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) clinical trials were turning studies away — they didn’t have the resources to test all of the new and potential advancements. But starting last year, the Great Ormond Street Hospital in…
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