February 2026

We are disappointed to learn the news that Roche has made the difficult decision to terminate their Phase 2 SHIELD DMD study evaluating satralizumab for bone health in Duchenne.  According to Roche, this decision was reached after careful consideration of the program’s path forward and reflects a combination of factors, including study timelines and the…

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Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their ability to swallow after receiving the gene therapy candidate BB-301 in an ongoing clinical trial. That’s according to an update from therapy developer Benitec Biopharma, which announced new data from the small trial (NCT06185673), which is…

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Charlotte is 11 years old and lives with LGMD2C, which was diagnosed when she was two and a half. When she grows up, Charlotte wants to get a law degree and become President so that she can make sure rare disease research is funded. Her biggest hope in life is that rare diseases are prioritized,…

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On Jan. 31, I visited the former Supreme Court Foyer at the National Gallery Singapore (NGS) for the final Artsplaining session of Light to Night Singapore 2026. NGS is a leading visual arts museum with the largest collection of Southeast Asian modern art. Artsplaining is its program designed to make the arts accessible to everyone.…

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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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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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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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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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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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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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