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FDA decision on DMD cell therapy pushed to November
The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from the Phase 3 HOPE-3 clinical trial (NCT05126758) and…
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Depression and muscular dystrophy
This story includes discussion of suicide. Support is available if you experience suicidal thoughts, are making plans to self-harm, feel like you or someone you know is unable to stay safe, or are in immediate danger that requires urgent help. If you or someone you know needs urgent mental health support, the following services are…
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Precision Biosciences Doses First Patient in FUNCTION-DMD Trial of PBGENE-DMD
Precision BioSciences, Inc. today announced the dosing of the first patient in the Phase 1/2 FUNCTION-DMD clinical trial, evaluating the safety, tolerability, and efficacy of PBGENE-DMD for the treatment of Duchenne. PBGENE-DMD is Precision’s investigational in vivo gene editing program designed to improve function by restoring near full-length dystrophin. PBGENE-DMD uses a single adeno-associated virus…
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Precision BioSciences has dosed the first individual with their novel gene editing approach for individuals with Duchenne mutations between exons 45 and 55
Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out exons 45-55 of the dystrophin gene, a therapeutic approach amenable to individuals with Duchenne with mutations in that region. Link to press release: https://investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-commences-dosing-phase-12-function-dmd Dear Duchenne…
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Maintaining ankle range of motion with muscular dystrophy
In the spring of 1986, my doctor told my parents and me that one of the standard prescribed treatments for muscular dystrophy was splints, which I would wear on my legs to keep my heel cords loose and limber. The doctors wanted my ankles to reach a 90-degree bend, so I wore thigh-high casts for…
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Capricor’s PDUFA date for FDA to review Deramiocel has been extended to November 22, 2026
Capricor Therapeutics announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne. The new PDUFA date is now November 22, 2026. After the FDA Advisory Committee Meeting in July, Capricor submitted an amendment to the…
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Update on Deramiocel: PDUFA Date Extended
Today, Capricor Therapeutics formally announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s amendment to the Biologics License Application (BLA) for Deramiocel, and the FDA will be extending the current Prescription Drug User Fee Act (PDUFA) action date of August 22, 2026. The FDA has classified the amendment as a major…
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This gut microbe may help keep you strong as you age
Scientists have linked a specific gut bacterium, Roseburia inulinivorans, to stronger muscles in both humans and mice. Older adults who carried it had 29% greater handgrip strength, while treated mice gained about 30% more grip strength and developed larger, more fast-twitch muscle fibers. The bacterium was also less common in older people. Researchers say it…
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Deramiocel: Where We Are Today
As we approach August 22, the FDA’s current Prescription Drug User Fee Act (PDUFA) action date for Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, PPMD recognizes how closely the Duchenne community is watching for an update. On August 13, 2026, Capricor shared the company’s plans to submit an amendment to the application, which the…
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From Research to Reality: Meet 2 Researchers Driving Drug Development Progress
Every therapy or intervention has a backstory. Long before a treatment reaches a clinic, it often begins with a grant that gives researchers the time and support to follow a promising idea. That path from lab to real life can take years, but it is how progress happens. The two researchers profiled here represent different…
