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Update on ELEVIDYS Shipments in the U.S.
This evening, Sarepta Therapeutics issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, effective close of business tomorrow, July 22, 2025. According to Sarepta, this pause will provide time for the company to respond to any requests for information and allow Sarepta…
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MDA Ambassador Guest Blog: Parenting, LGMD, and Choosing Joy
Chris Carroll is 41 years old and lives with his wife Joy and two children Jordyn (4) and Brogan (who will be 3 soon). Chris works for Mullen Coughlin LLC. A data privacy law firm in Devon Pa. In his free time, Chris loves going to Phillies games with his family and going out to…
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ELEVIDYS UPDATE
Read Sarepta’s letter to the community Shared July 18, 2025 Duchenne Community Letter 7.18.2025Download The FDA has revoked Sarepta’s platform technology designation and requested that Sarepta voluntarily stop all shipments of ELEVIDYS today. Sarepta has issued a statement indicating that they will continue to ship ELEVIDYS to the ambulant population. Read FDA press release HERE…
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As I look in the mirror, I’m proud of the person staring back at me
Years ago, I had my colors done: I’m a winter. Dark blues, greens, scarlets, royal hues, red, black, and hot pink look best on me. I don’t always follow these guidelines, but I own a few statement pieces in these shades and wear them when I feel confident. On Tuesday, I wore a hot pink…
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ELEVIDYS Update
PPMD acknowledges the media reports that the U.S. Food and Drug Administration (FDA) will request Sarepta Therapeutics stop shipping ELEVIDYS for all Duchenne patients. These reports are profoundly upsetting and raise serious concerns for our entire community. Families who have fought tirelessly for access to this therapy, those who have already received it, and those…
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Sarepta Therapeutics Announces Update on ELEVIDYS Label, Company Restructuring
Yesterday afternoon, Sarepta issued a press release and community letter which included updates related to the non-ambulatory label for ELEVIDYS. While ELEVIDYS continues to be available for individuals living with Duchenne who are ambulant, Sarepta paused shipments for non-ambulant patients following a second safety event in June. Key takeaways for the Duchenne community from this…
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Dosing begins in gene therapy trial for DMD-linked heart disease
The first patient has been dosed as part of a Phase 1b clinical trial of AAV1.SERCA2 (SRD-003), Sardocor‘s one-time gene therapy for cardiomyopathy — a type of heart disease — associated with Duchenne muscular dystrophy (DMD-CM). The multicenter trial, dubbed MUSIC-DMD (NCT06224660), is being conducted in collaboration with the University of Kansas Medical Center. It’s…
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New preclinical data from Precision BioSciences’s gene editing program for Duchenne
Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in the “hot spot” region of exons 45-55, with the goal of submitting applications later this year to begin clinical trials in the US and/or Canada. Precision’s technology is a gene editing approach, which aims to permanently alter an individual’s own…
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With FSHD, dealing with pain is a daily challenge for me
In a daily devotional I’m reading titled “Incurable Faith: 120 Devotions of Lasting Hope for Lingering Health Issues,” author Andrea Herzer describes pain as a thief, stealing her joy. Her words ring true for me as I navigate life with facioscapulohumeral muscular dystrophy (FSHD) and other health issues. Relentless discomfort is trying to steal my…
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First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy
Medera, and its clinical division Sardocor, announced that the first participant was dosed with AAV1.SERCA2a in a Phase 1b clinical trial for cardiomyopathy secondary to Duchenne. Cardiac complications are the leading cause of mortality in Duchenne, and this experimental gene therapy delivers the SERCA2a gene with the goal of correcting the calcium dysregulation in cardiac…
