An investigational gene therapy called GNT0004 has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial…
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The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 to 9 years old, according to new trial analyses. Boys with DMD at this age are typically expected to show…
Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval. The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The…
Parent Project Muscular Dystrophy (PPMD), a nonprofit dedicated to ending Duchenne muscular dystrophy (DMD), has published an updated edition of the PJ Nicholoff Steroid Protocol, marking the 10th anniversary of its first release. Called PJ’s Protocol, the guidance is designed to help healthcare providers manage corticosteroid use…
Solve FSH, a venture philanthropic organization, has invested $3 million to support the development of Armatus Bio‘s microRNA-based therapy ARM-201 as a treatment for facioscapulohumeral muscular dystrophy (FSHD). ARM-201 is designed to block the production of DUX4, the protein that is abnormally produced in people with FSHD, a…
The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for…
People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease, according to a U.S. study that analyzed a large database of insurance claims. After diagnosis, DM patients are more likely to be hospitalized and visit an emergency department, researchers found.
The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk. The European…
CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and…
It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S. Since its…
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