Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School of Medicine to advance research into a mini-dystrophin gene therapy that may protect cardiac function in Duchenne muscular dystrophy (DMD) patients. The funds were raised during PPMD’s Cardiac Initiative holiday campaign highlighting the…
G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society define as including Dravet syndrome. All three designations are among some 70,000 diseases listed in the latest…
Severe Duchenne muscular dystrophy (DMD) is linked to increased susceptibility to a rare and aggressive type of muscle cancer called rhabdomyosarcoma, a mouse study suggests. The finding was reported by researchers from Sanford Burnham Prebys Medical Discovery Institute in the study, “Muscle Stem Cells…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
Popeyes Louisiana Kitchen’s franchisee Sailormen has launched the 15th Annual “Appetite for a Cure” campaign to enhance the lives of those living with muscular dystrophy (MD) and related disorders. Money raised will support efforts by the Muscular Dystrophy Association (MDA) to combat MD and other diseases of the…
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…
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