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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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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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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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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
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Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP)
Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and
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FDA designation highlights new treatment approach for DMD
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to (Z)-endoxifen for Duchenne
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Navigating College Scholarships and Financial Assistance with a Disability
Like many high school students, students with neuromuscular diseases may be looking at college as
