News

Catabasis Pharmaceuticals recently completed its target enrollment of 30 patients for the open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (DMD). Edasalonexent is an oral drug that researchers hope will revolutionize DMD treatment. Edasalonexent…

PTC Therapeutics‘ new investigational therapy ataluren shows promise for preserving lung function and slowing disease progression in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients, according to a recent news release. The results came from an analysis comparing lung function data from a PTC extension study (Study 019; NCT01557400) and data…

Nonprofit organizations CureDuchenne and Art In Giving are co-hosting a “Dealing for Duchenne” charity event in Cambridge, Massachusetts, on Oct. 20 to support the quest for a Duchenne muscular dystrophy (DMD) cure. Cambridge will emulate vintage Vegas for a one-off night featuring poker, blackjack, and other classic…

Translarna (ataluren) is a promising drug for the treatment of Duchenne muscular dystrophy (DMD) and cystic fibrosis. A team of scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham has provided additional knowledge into Translarna’s mechanism of action, advancing our understanding of how the drug works to…

Using a newly engineered tongue-on-a-chip model of Duchenne muscular dystrophy, researchers found one reason that muscle regeneration fails — muscle stem cells react poorly to signals telling them new muscles need to be formed. The study,  “A human in vitro model of Duchenne muscular dystrophy muscle formation and contractility,” published in the…

Elixirgen has announced the launch of its new Quick-Tissue 1.0 Series for use by scientists exploring new ways of treating muscle-degenerative diseases like muscular dystrophy (MD). The biopharmaceutical company, located at Johns Hopkins campus, specializes in stem cell biology. The Quick-Tissue 1.0 Series includes a skeletal muscle differentiation kit called…

Researchers from Saudi Arabia demonstrated that a neurological panel assay that they developed, offers a high diagnostic yield for limb girdle muscular dystrophy (LGMD) and other myopathies.

Idera Pharmaceuticals recently presented preclinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentations took place at the recent 12th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal,…

Summit Therapeutics announced that it has received a Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for its lead drug candidate, ezutromid, as a treatment for Duchenne muscular dystrophy (DMD). The FDA defines Rare Pediatric Diseases as disorders that primarily affect people from birth to age…

Capricor Therapeutics, Inc. announced that the U.S. Department of Defense (DoD) has awarded the company a $2.4 million grant to establish a scalable, commercially-ready process to manufacture CAP-2003, the company’s proprietary exosome product candidate. Capricor is a clinical-stage biotechnology company working on the discovery, development and commercialization of biological therapies.