Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen) may help protect the heart as well as muscles used for movement, a study found. The treatment slowed heart function decline, raising the possibility it could lower the risk of cardiomyopathy, a serious condition that can lead to heart failure.
Across the U.S., the neuromuscular disease community is preparing to mark Muscular Dystrophy Awareness Month this September, with a series of events and activities aimed at spotlighting muscular dystrophy (MD) and other related conditions. These efforts, organizers say, also seek to raise vital funds to support all individuals in…
Levels of certain proteins implicated in immune response and the body’s process to stop bleeding differ between Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), according to a study Also, blood levels of these proteins correlated with the degree of motor function in BMD participants, but not…
Researchers have developed a detailed molecular model for the structure of type VI collagen and identified where genetic mutations linked to certain forms of muscular dystrophy (MD) might influence the protein. Findings from their study could ultimately help scientists better understand the mechanisms underlying types of MD that affect…
Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with myotonic dystrophy type 1 (DM1), according to an announcement from Sarepta Therapeutics. The trial recently hit one of two prespecified enrollment targets, prompting a review of safety data.
The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders…
A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may…
Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of facioscapulohumeral muscular dystrophy (FSHD), a disease type in which symptoms like muscle weakness typically become apparent by adulthood. The Phase 1/2 clinical trial (NCT06907875) is expected to enroll…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation is intended to accelerate the development and regulatory review of medications intended to treat serious…
An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences has received orphan drug status from the U.S. Food and Drug Administration (FDA). The designation focuses on treatments for rare diseases like DMD. Its benefits include tax credits and fee exemptions, along with…
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