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Guest Voice: Celebrating World Collagen 6 Myopathy Day
I know what you’re thinking: another awareness day. One more health observance to briefly acknowledge
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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
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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
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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
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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
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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
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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
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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
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Sarepta Webinar
ELEVIDYS Gene Therapy Update and ENDEAVOR Cohort 8 Awareness Watch HERE Recorded: May 19, 2024
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Tevard Webinar
Unlocking the therapeutic power of suppressor tRNAs for DMD Watch HERE Recorded Thursday, May 28,
