A potential next-generation therapy, golodirsen (SRP-4053) facilitates and increases dystrophin production in Duchenne muscular dystrophy (DMD) patients, an interim analysis of a Phase 1/2 trial being presented at the 2018 American Academy of Neurology (AAN) Annual Meeting in Los Angeles reports. These findings, in “Golodirsen Induces Exon Skipping Leading…
A stem cell therapy improved the heart function, walking capacity and survival of mice with Duchenne muscular dystrophy, a study reports. The treatment involves what scientists call cardiosphere-derived cells, or CDCs. They are the research equivalent of the stem cell therapy known as CAP-1002 that Capricor Therapeutics is developing to treat…
Researchers found that giving Duchenne muscular dystrophy (DMD) patients glucocorticoid therapy keeps them mobile for a longer period of time and reduces their risk of dying. Their study, “Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study,”…
•The Muscular Dystrophy Association has awarded 13 grants totaling $3.5 million to researchers working on treatments for neuromuscular diseases such as MD and ALS, studies that identify the diseases’ causes, or research on the disorders’ underlying mechanisms. This brings the total amount of the grants the organization has made this…
Research into molecules that appear to target the gene known to cause facioscapulohumeral dystrophy (FSHD) may lead to a treatment able to slow disease progression, a study reports. The study, “BET bromodomain inhibitors and agonists of the beta-2 adrenergic receptor identified in screens for compounds that inhibit DUX4 expression…
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