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Roche Announces Termination of Phase 2 SHIELD DMD Study
We are disappointed to learn the news that Roche has made the difficult decision to
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New gene therapy for OPMD shows lasting success in small US trial
Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced
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MDA Ambassador Guest Blog: The Secret to Navigating Life with a Rare Disease? Say Yes.
Charlotte is 11 years old and lives with LGMD2C, which was diagnosed when she was
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How art fosters a sense of belonging in my life with Duchenne
On Jan. 31, I visited the former Supreme Court Foyer at the National Gallery Singapore
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Entrada to initiate an increased dose cohort in their Duchenne exon-44 skipping program
As an early funder of Entrada Therapeutics, CureDuchenne is pleased to share that after the
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1st boys dosed in Phase 2 trial of oral DMD treatment SAT-3247
Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in
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Everything You Need to Know About the Upcoming 2026 MDA Clinical & Scientific Conference
The 2026 MDA Clinical & Scientific Conference is quickly approaching. Taking place March 8-11, 2026,
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Guest Voice: Despite losses from Duchenne MD, I persevere
I was born in March 1988 with the umbilical cord wrapped around my ankles three
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I rarely answer honestly when asked, ‘How are the boys doing?’
It’s been a busy couple of weeks. There have been lots of appointments and activities,
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PTC Therapeutics Provides Regulatory Update and Withdraws Its New Drug Application for Translarna™
We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for
