Microdystrophin gene therapy robustly induces the production of a shorter, but functional, version of the dystrophin protein and reduces muscle damage in Duchenne muscular dystrophy (DMD) patients, according to preliminary results of a Phase 1/2 clinical trial. DMD is a severe type of muscular dystrophy caused by mutations in the…
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, a dystrophin gene therapy aiming to treat Duchenne muscular dystrophy (DMD). FDA, in its letter, said that Solid Biosciences, the therapy’s developer, had addressed all questions of concern that led to…
The Centers for Disease Control and Prevention (CDC) released new, updated guidelines for the management of Duchenne muscular dystrophy (DMD) patients. Researchers highlighted the role of rehabilitation techniques and the importance of physiatrists to improve function and quality of life. The study,…
Researchers identified proteins that regulate DUX4, the key gene in facioscapulohumeral muscular dystrophy (FSHD). The results offer a potential therapeutic pathway, they believe. The study, “NuRD and CAF-1-mediated silencing of the D4Z4 array is modulated by DUX4-induced MBD3L proteins,” appeared in the journal eLife. While healthy…
Ryan Getzlaf is hosting the 8th Annual Getzlaf Golf Shootout Sept. 8 to raise funds for CureDuchenne and its mission to find a cure for Duchenne muscular dystrophy (DMD). Getzlaf is the captain of the Anaheim Ducks of the National Hockey League. The two-day charity golf event will bring…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA…
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51 of the dystrophin gene, the treatment’s developer, Sarepta Therapeutics, has announced. The company…
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…
Getting your son into a Duchenne muscular dystrophy (DMD) clinical trial can be difficult, given the narrow criteria for eligibility — like walking ability — associated with such trials. Even for families who qualify, the experience can be draining, said Pat Furlong, founder and CEO of Parent Project Muscular…
The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended expanding the use of Translarna (ataluren) to ambulatory children ages 2-5 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD). CHMP’s recommendation of Translarna use in younger children is based…
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