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GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in Muscular Dystrophy conference, held in Chicago earlier…

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The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular dystrophy, our desire to control the course of events can be a creative balancing act. We hope it’s like riding a…

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Life feels busy, which is no surprise to me. My husband, Jason, and I share seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (DMD). Our lives are busy year-round. This year seems especially hectic because I returned…

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Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could support the therapy’s accelerated approval by the U.S. Food and Drug Administration (FDA). Regenxbio has also announced new data showing RGX-202 treatment led to physical…

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On Saturday, I presented at the Participation — Inclusion in Action Conference 2024, which was held in my home of Singapore for the first time. My physiotherapist at the National University Hospital, who’s also a disability and healthcare researcher and one of the event’s key organizers, invited me to participate in February. In my talk,…

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REGENXBIO recently joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, REGENXBIO’s microdystrophin gene therapy. Dr. Naz Dastgir discussed the company’s pivotal trial plans and shared interim Phase I/II clinical data, including the first functional data for RGX-202. The webinar also included an extensive Q&A portion, during…

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PPMD commemorated its 30th anniversary with the “Investing in Every Future” celebratory event on November 14, 2024, in New York City. With some 200 attendees, this meaningful gathering honored three decades of progress and the resilience and spirit of the Duchenne community, and demonstrated the organization’s commitment to fighting for every future as PPMD continues to…

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A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD), according to a study. The results have potential implications for gene therapies. The technology works by delivering two halves of these large genes separately, using harmless…

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CureDuchenne is delighted to share the positive updates from REGENXBIO on their Phase 1/2 open-label clinical trial of RGX-202, an AAV8-delivered microdystrophin with an extended C-terminal domain.  RGX-202 demonstrated robust microdystrophin expression at both dose levels, as well as functional improvements in individuals (aged 4-11) treated compared to natural history controls.  REGENXBIO has expanded the…

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REGENXBIO Inc. has announced that the AFFINITY DUCHENNE® open-label trial of RGX-202 has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data. RGX-202 utilizes a new, modified  adeno-associated virus (AAV8) to transport…

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