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.
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 MoveÂ鶹Éç clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (Â鶹Éç). Edasalonexent is an oral drug that researchers hope will revolutionize Â鶹Éç 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 (Â鶹Éç) 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 (Â鶹Éç) 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 (Â鶹Éç). The FDA defines Rare Pediatric Diseases as disorders that primarily affect people from birth to age…
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