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Massachusetts Begins Newborn Screening for Duchenne
As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor Maura Healey on August 23, 2024. The law included a critical amendment funding the program and mandating the addition of…
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Dyne hopes to bring new treatment for DM1 to US market in 2028
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen (z-basivarsen), an experimental treatment for myotonic dystrophy type 1 (DM1). The company announced that enrollment is now complete for this key group of patients. If the results are positive, Dyne plans to apply for accelerated approval…
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Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027
Read Avidity’s Community Letter: Avidity Community Letter_DMD45 Program Update_June 2026Download The post Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027 appeared first on CureDuchenne.
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MDA Ambassador Guest Blog: My Myasthenia Gravis Voyage
Tina Vassar lives in Durham, North Carolina with her dog Milo. She is 69 years young and has lived with myasthenia gravis for over 30 years. Like many people living with myasthenia gravis, I have a unique story – or, as I call it, my voyage. Tina Vassar I was diagnosed with anti-ACHR+ generalized myasthenia…
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PPMD Joins FDA Leadership for Rare Disease Roundtable
On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of the Rare Disease Innovation Hub on behalf of Acting Commissioner Kyle Diamantas, JD, Acting Director of CBER, Karim Mikhail, B.…
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Entrada shares updates on Duchenne programs, including initiation of Cohort 2 in UK and EU trial skipping exon 45
As an early funder of Entrada Therapeutics, we are pleased to share their latest newsletter to the Duchenne community. This update includes news that after review of all the data in the first dose (5 mg/kg) cohort of the ELEVATE-45-201 study for individuals amenable to skipping exon 45, an independent data monitoring committee has recommended…
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Adults with MD report major gaps in sexual, reproductive healthcare: Study
Adults with muscular dystrophy (MD) in the U.S. report widespread gaps in sexual and reproductive healthcare, according to surveys and interviews. Such gaps included a lack of private clinical time, provider discomfort, and unaddressed concerns about fertility and relationships. At the same time, participants indicated a strong desire for proactive, disability-informed guidance from their medical…
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Sarepta Webinar
ELEVIDYS Gene Therapy Update and ENDEAVOR Cohort 8 Awareness Watch HERE Recorded: May 19, 2024 The post Sarepta Webinar appeared first on CureDuchenne.
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Tevard Webinar
Unlocking the therapeutic power of suppressor tRNAs for DMD Watch HERE Recorded Thursday, May 28, 2026 The post Tevard Webinar appeared first on CureDuchenne.
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Regenxbio Webinar
RGX-202: Investigational Gene Therapy for Duchenne Muscular Dystrophy Pivotal Data Update from the AFFINITY DUCHENNE® Study Watch HERE Recorded Tuesday, May 19, 2026 The post Regenxbio Webinar appeared first on CureDuchenne.
