The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a…
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Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global clinical trial able to walk. That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood — that’s now being tested in a clinical trial involving DM1 patients. This status is awarded by the…
Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was given once weekly for six months to 19…
The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer Dyne Therapeutics. The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants…
Note: This story was updated Jan. 17, 2025, to clarify the company’s comments on gene therapies, which generally deliver a smaller form of dystrophin. The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people…
A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and help regenerate muscle tissue in a mouse model of the disease. The researchers are now seeking…
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD). “The submission of the BLA marks a pivotal step for Capricor and those impacted by…
During 2024, Muscular Dystrohy News Today covered the latest scientific studies, advances in treatment, and clinical trials for the multiple types of muscular dystrophy (MD). Here is a list of the top 10 most-read stories we published last year. No. 10 – Patient death prompted pause in DMD…
The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases. Altogether, the funding totals more than $5 million across 21 grants, according to a press release…
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