The nonprofit Parent Project Muscular Dystrophy (PPMD) has awarded $239,000 to Dr. Terence Partridge, with the Research Center for Genetic Medicine at Children’s National Medical Center. The grant will support ongoing work aimed at a better understanding of exon skipping therapies for Duchenne muscular dystrophy (DMD), and their validation in future clinical trials. DMD is caused by mutations in…
Pain is a common and frequent problem among patients with Duchenne muscular dystrophy (DMD) and is associated with physical limitations and poor quality of life in these patients, according to a new study conducted by researchers in Brazil. The authors argue that knowing several aspects about pain, such as frequency,…
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…
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