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MDA Ambassador Guest Blog: Grateful for the One by My Side on Valentine’s Day (and Every Day)
Jeff Thomas resides in beautiful Boise, Idaho with his partner in crime, Christine, and their precious fur-babies, Tito, Flora, and Fred. Their love and support motivate him in the fight against oculopharyngeal muscular dystrophy OPMD and empower him to advocate for everyone living with neuromuscular disease. Me and the girl in the white leather jacket.…
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Trial testing gene-editing therapy PBGENE-DMD gets FDA green light
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing therapy candidate PBGENE-DMD in people with Duchenne muscular dystrophy (DMD) caused by certain mutations. Precision announced it had received a “Study May Proceed notification” from the U.S. regulatory agency that will allow clinical testing of the…
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Satellos, a CureDuchenne funded company, just announced the first participant was dosed in their Phase 2 pediatric study of SAT-3247 for Duchenne muscular dystrophy.
Read news release HERE The post Satellos, a CureDuchenne funded company, just announced the first participant was dosed in their Phase 2 pediatric study of SAT-3247 for Duchenne muscular dystrophy. appeared first on CureDuchenne.
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PTC Therapeutics Provides Regulatory Update on Translarna™ (atalauren)
My heart is heavy today following PTC’s decision to withdraw their NDA submission for Atalauren (Translarna). PTC submitted their package to FDA in 2024, based on FDA’s commitment to review the totality of evidence for Atalauren, a small molecule for individuals with nonsense variants. PTC’s decision is based on recent discussions with FDA during which…
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A winter blizzard had me cooped up at home for days
Pittsburgh, where I now live, receives far less annual snowfall than Shanksville, Pennsylvania, where my wife, Wendy, and I lived for 41 years. Shanksville, in Somerset County, sits squarely in the state’s snow belt. However, every so often, Pittsburgh experiences true winter weather, with bitter cold and significant snowfall. Just a couple of weeks ago,…
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Recent Updates with Section 504
In January, nine states led by Texas revised their challenge to Section 504 of the Rehabilitation Act and the integration mandate required by both Section 504 and the Americans with Disabilities Act (ADA) by arguing in an amended complaint that the mandate, which requires states to fund services in the most integrated setting, is unconstitutional. The Rehabilitation Act, including Section 504, is a landmark…
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Rare Disease Advocacy Delivers: Major Federal Wins for Duchenne and Becker Community
We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers. With the signing of the FY 2026 Consolidated Appropriations Act (H.R. 7148) on February 3, 2026, Congress enacted a powerful package…
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Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND)
Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy. This clearance allows the company to move forward with a Phase 1/2 clinical study to evaluate safety and early signals of effectiveness. The investigational therapy uses Precision’s ARCUS® gene editing platform and…
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New Phase 3 trial of Duchenne gene therapy SGT-003 to begin dosing
A Phase 3 trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will start dosing before the end of March, according to Solid Biosciences. The trial follows a successful meeting with the U.S. Food and Drug Administration (FDA), during which the company and the agency aligned on the study’s design. The IMPACT DUCHENNE…
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CureDuchenne invests in Satellos as it raises $57.2M to advance their Duchenne program through clinical trials
CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne TherapyLink to press release CureDuchenne is pleased to announce that it has participated in the Satellos Bioscience public offering, supporting the company’s $57.2 million financing to advance its Duchenne muscular dystrophy program through clinical development. The funds will support ongoing and planned clinical trials of SAT-3247,…
