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Entrada to initiate an increased dose cohort in their Duchenne exon-44 skipping program
As an early funder of Entrada Therapeutics, CureDuchenne is pleased to share that after the independent Data Monitoring Committee reviewed the safety data from Cohort 1 of the ELEVATE-44-201 trial in individuals with Duchenne amenable to skipping exon 44, Entrada received recommendation to initiate the higher dose Cohort 2 at 12 mg/kg. Entrada expects to…
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1st boys dosed in Phase 2 trial of oral DMD treatment SAT-3247
Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular dystrophy (DMD). The study, dubbed BASECAMP (NCT07287189), aims to enroll 51 boys with DMD aged 7-9 who can walk. Patients who have received treatments such as exon skippers, corticosteroids, Duvyzat (givinostat), and Elevidys (delandistrogene moxeparvovec-rokl) may be…
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Everything You Need to Know About the Upcoming 2026 MDA Clinical & Scientific Conference
The 2026 MDA Clinical & Scientific Conference is quickly approaching. Taking place March 8-11, 2026, at the Hilton Orlando in Orlando Florida, this year’s conference will explore the latest research and clinical advancements for neuromuscular disease. In an era of new treatments and life-changing discoveries, this gathering of leading clinicians, researchers, industry partners, advocacy organizations…
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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…
