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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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, […]
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Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee
Have you been looking for a way to engage with the community more? Do you […]
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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 […]
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The missed milestones in the teenage years because of DMD
I have three teenage sons — Max, 18, Rowen, 15, and Charlie, 13 — who […]
