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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 […]
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Patients with certain mutations lose their walking ability earlier: Study
Specific genetic mutations in Duchenne muscular dystrophy (DMD) influence how long patients retain the ability to […]
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Solid Biosciences Shares Updates on INSPIRE DUCHENNE Clinical Trial of SGT-003
Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation […]
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REGENXBIO Shares Updates on Phase I/II AFFINITY DUCHENNE® trial of RGX-202
Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for […]
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I always try to pause to appreciate the small victories
I’m currently continuing to do the exercises my physical therapist recommended during my last therapy […]
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Supporting artists with disabilities through a new business venture
Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding […]
