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MDA Ambassador Guest Blog: Grateful for the One by My Side on Valentine’s Day (and Every Day)
Jeff Thomas resides in beautiful Boise, Idaho with his partner in crime, Christine, and their
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Trial testing gene-editing therapy PBGENE-DMD gets FDA green light
The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical
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Satellos, a CureDuchenne funded company, just announced the first participant was dosed in their Phase 2 pediatric study of SAT-3247 for Duchenne muscular dystrophy.
Read news release HERE The post Satellos, a CureDuchenne funded company, just announced the first
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PTC Therapeutics Provides Regulatory Update on Translarna™ (atalauren)
My heart is heavy today following PTC’s decision to withdraw their NDA submission for Atalauren
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A winter blizzard had me cooped up at home for days
Pittsburgh, where I now live, receives far less annual snowfall than Shanksville, Pennsylvania, where my
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Recent Updates with Section 504
In January, nine states led by Texas revised their challenge to Section 504 of the Rehabilitation Act
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Rare Disease Advocacy Delivers: Major Federal Wins for Duchenne and Becker Community
We applaud a major legislative victory for the rare disease and Duchenne and Becker community
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Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND)
Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application
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New Phase 3 trial of Duchenne gene therapy SGT-003 to begin dosing
A Phase 3 trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will
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CureDuchenne invests in Satellos as it raises $57.2M to advance their Duchenne program through clinical trials
CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne TherapyLink to press release CureDuchenne is
