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Guest Voice: Despite losses from Duchenne MD, I persevere
I was born in March 1988 with the umbilical cord wrapped around my ankles three times. My heart rate dropped to zero, and the doctors had to do an emergency cesarean section to save me. I still reached all my milestones like a normal baby. When I was 6 years old, my teacher noticed I…
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I rarely answer honestly when asked, ‘How are the boys doing?’
It’s been a busy couple of weeks. There have been lots of appointments and activities, and I’ve been busy running errands around town. I live in a rural community in south central Nebraska. It’s small enough that, no matter where I go, I run into someone I know. I also have a very large family,…
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PTC Therapeutics Provides Regulatory Update and Withdraws Its New Drug Application for Translarna™
We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the data are insufficient to meet the standard for approval. The company is expected to inform the community in the coming weeks of next steps for…
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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…
