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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
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Insights by Ira: Mission Complete
Just four days prior to my inaugural public engagement as National Ambassador in 2024, I
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Dyne, citing positive trial data, to ask FDA to approve DMD therapy
Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to
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My hope for the FSHD community this Advent season
Advent is the beginning of the church year for Christians. What better way to start
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Dyne announces positive topline results from Phase 1/2 trial of z-rostudirsen in Duchenne amenable to skipping exon 51
As an early investor in Dyne Therapeutics, we are pleased to share their exciting news
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New trial results highlight gains in arm and heart function for DMD
In the Phase 3 HOPE-3 clinical trial, the experimental cell therapy deramiocel outperformed a placebo
