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WATCH: RGX-202 – AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording)
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 Culminates 30th Anniversary with Celebratory “Investing in Every Future” Event
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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New technology may inform effective MD gene therapies: Study
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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Positive data reported for REGENXBIO’s AAV8-delivered microdystrophin, RGX-202
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 Initiates Pivotal Phase of AFFINITY DUCHENNE® Trial of RGX-202 Gene Therapy, Reports Positive Functional Data
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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Being mindful of kidney stones while living with DMD
“Kidney stones!” my 13-year-old son, Charlie, exclaimed when I asked him what I should write about this week. My husband, Jason, and I have seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 15; Charlie, Mary, 10; and Callie, 2. Max, Rowen, and Charlie have Duchenne muscular dystrophy (DMD). I decided to take up Charlie’s…
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FDA OKs trial to test gene therapy candidate in LGMD children
The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical trial to test ATA-200 — the company’s gene therapy candidate for limb-girdle muscular dystrophy type 2C/R5, or LGMD2C/R5 — in children with this form of LGMD in the U.S. The milestone means that ATA-200 will be…
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New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
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Coping with grief and the day-to-day life of LGMD
People living with a chronic illness, including me, commonly explore how we deal with grief, which is the subject of much theory and research. I’ve thought deeply about how living with limb-girdle muscular dystrophy (LGMD) can be seen through the familiar Kübler-Ross model of the grief cycle, with its stages of denial, anger, bargaining, depression,…
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Sarepta stops development of exon 51-skipping therapy for DMD
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), an exon 51-skipping therapy for some people with Duchenne muscular dystrophy (DMD). While increases in dystrophin levels seen with the exon-skipping treatment were encouraging, according to the company, concerns over the long-term safety and tolerability of SRP-5051 influenced the…
