The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development…
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Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type…
CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing gene therapies. “This investment underscores our continued use of venture philanthropy to catalyze progress [toward] transformative treatments…
Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO…
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…
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…
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