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Watch: Capricor Therapeutics — HOPE-3 Community Update (Webinar Recording)
PPMD recently held a community webinar with Capricor Therapeutics, during which the company shared their
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BREAKING NEWS: U.S. Department of Health and Human Services (HHS) adds Duchenne Muscular Dystrophy to the Recommended Uniform Screening Panel (RUSP) for Newborn Screening
CureDuchenne applauds the U.S. Department of Health and Human Services (HHS) for adding Duchenne muscular dystrophy to
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Dyne Webinar: Positive Topline Results from DELIVER trial in Exon 51 DMD
A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares
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Clinical Research Alert: Phase 1 Study of Gene Therapy in Individuals with Myotonic Dystrophy Type 1
Researchers at Sanofi are seeking individuals with non-congenital myotonic dystrophy type 1 (DM1) to participate in
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Reflecting on 2025’s Advocacy Accomplishments
The last 12 months have been full of twists and turns in Washington D.C., but
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Advocates cheer federal push to test every newborn for Duchenne
Every newborn in the United States should now be screened for Duchenne muscular dystrophy (DMD),
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A Shift in Perspective: Person-First Language and Identity-First Language
When the disability rights movement began in the 1970s, the disability community started forging the
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Clinical Research Alert: Phase 1/2 Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1
Researchers at Dyne Therapeutics, Inc. are seeking individuals with myotonic dystrophy type 1 (DM1) to participate
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Clinical Research Alert: Two Phase 4 Studies of Risdiplam in Pediatric SMA Patients Following Gene Therapy
Researchers at Genentech Inc. are seeking pediatric patients previously treated with gene therapy (onasemnogene abeparvovec) for
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Reflecting on another year of living with DMD
This column has followed me through a year that has asked a lot of my
