Resolaris, a potential intravenous protein therapy developed by aTyr Pharma for the treatment of rare myopathies is generally safe and well tolerated in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) according to results presented at the 21st International Congress of the World Muscle Society.
A gene called CD40, which is an important modulator of the immune system, may determine the severity of Duchenne muscular dystrophy (DMD) according to a study published in The American Journal of Human Genetics. This finding suggests that drugs targeting CD40 may improve the symptoms of DMD.
People with Duchenne muscular dystrophy are most anxious and worried about their condition when they have to transition to using a wheelchair or a respirator due to the progression of their condition, found a study published in the International Journal of Qualitative Studies on Health and Well-being.
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.
Get regular updates to your inbox.
