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Dyne initiates global Phase 3 trial in Duchenne amenable to skipping exon 51, ahead of planned FDA submission for accelerated approval in the US
Dyne Therapeutics, which received early funding from CureDuchenne, is initiating a Phase 3 FORZETTO Trial of z-rostudirsen in individuals with Duchenne amenable to skipping exon 51. Dyne is on track to submit a BLA to the FDA for accelerated approval in the US later this quarter, and the FORZETTO trial is intended to serve as…
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In my life with DMD, I want to keep paying it forward, one story at a time
This week, I found myself reflecting on how much my life has changed since I joined Shalom Medcare, a care transport and medical escort provider in Singapore that was founded in 2021. Working there has become a part of how I advocate for healthcare equality for those who live with Duchenne muscular dystrophy (DMD), as…
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CureDuchenne Announces Partnership with Tevard Biosciences to Advance Suppressor tRNA Therapy for the Treatment of Duchenne Muscular Dystrophy
Tevard Biosciences to present most recent data to Duchenne community for first time at CureDuchenne FUTURES National Conference on May 22 with webinar to follow on May 28 Newport Beach, Calif., May 20, 2026 —CureDuchenne, a global leader in funding and advancing research for Duchenne muscular dystrophy, today announced a second investment into Tevard Biosciences…
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Dyne Announces Phase 3 Trial of Z-Rostudirsen, Plans for Submitting Biologics License Application to FDA
Dyne Therapeutics today announced the initiation of the company’s Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251), in individuals living with Duchenne who are amenable to exon 51 skipping. Z-rostudiresen is an investigational exon skipping therapy bound to an antigen-binding fragment (Fab) to help the therapy reach the muscle cells for those amenable to…
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CureDuchenne Appoints Lidia Gardner, PhD, EMBA, as Vice President of Clinical and Medical Affairs to Advance Patient-Centered Care, Research, and Strategic Partnerships
NEWPORT BEACH, Calif., May 20, 2026 /PRNewswire/ — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of Lidia Gardner, PhD, EMBA, as its new Vice President of Clinical and Medical Affairs. Dr. Gardner brings more than 15 years of experience spanning clinical strategy, patient advocacy, evidence generation,…
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RGX-202 gene therapy meets main goal in Phase 3 DMD clinical trial
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a press release. Steve Pakola, MD, Regenxbio’s chief medical officer, said the data “support the potential of RGX-202 to become a best-in-class gene therapy for Duchenne…
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When it comes to raising a child with DMD, the third time is not the charm
They say the third time is the charm. However, as a mom to three sons with Duchenne muscular dystrophy (DMD), I don’t find that to be the case. I share seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie…
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NEW: Livestream with the PAAC
What can you learn from the PPMD Adult Advisory Committee (PAAC) that you can’t ask Google? Honestly? A lot. Google can give you information, but it can’t tell you what it’s actually like to live with Duchenne or Becker muscular dystrophy. It can’t share the little workarounds, the “learned the hard way” moments, or the tips…
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MDA taps Bridgebio funding to improve LGMD care coordination
Bridgebio is providing $100,000 to the Muscular Dystrophy Association (MDA) to advance projects aimed at improving multidisciplinary care for people with limb-girdle muscular dystrophy (LGMD). The awards will go to initiatives at Stanford Health Care and the University of Minnesota that aim to make LGMD care more coordinated, accessible, and flexible for patients across the…
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RegenXBio announces positive topline results from pivotal Phase 3 microdystrophin gene therapy trial
RegenXBio shared positive topline results from their pivotal Phase 3 AFFINITY DUCHENNE study of RGX-202 in 31 individuals with Duchenne aged 1 year and older. The study met the primary endpoint with statistical significance, with 93% of participants achieving microdystrophin expression above 10%. There was also a statistically significant correlation between microdystrophin expression and functional…
