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Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027
Read Avidity’s Community Letter:
Regenxbio Webinar
RGX-202: Investigational Gene Therapy for Duchenne Muscular Dystrophy Pivotal Data Update from the AFFINITY DUCHENNE® Study Watch HERE Recorded Tuesday, May 19, 2026
Tevard Webinar
Unlocking the therapeutic power of suppressor tRNAs for DMD Watch HERE Recorded Thursday, May 28, 2026
Sarepta Webinar
ELEVIDYS Gene Therapy Update and ENDEAVOR Cohort 8 Awareness Watch HERE Recorded: May 19, 2024
CureDuchenne Appoints Lidia Gardner, PhD, EMBA, as Vice President of Clinical and Medical Affairs to Advance Patient-Centered Care, Research, and Strategic Partnerships
NEWPORT BEACH, Calif., May 20, 2026 /PRNewswire/ — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, proudly announces the appointment of Lidia Gardner, PhD, EMBA, as […]
First Participant Dosed in Phase 3 Trial for SGT-003 in Duchenne
Solid Biosciences has announced the first participant has been dosed in the Phase 3 IMPACT DUCHENNE trial evaluating SGT-003. The global study is now underway, with active sites in Australia […]
STAT: A decade ago, these drugs tore apart the FDA. Today might be some patients’ best hope.
By Jason Mast
Ataluren Update for the Duchenne Community
PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA). The update notes that a limited remaining supply […]
Solid Biosciences Provides Positive Interim Clinical Update from Phase 1/2 INSPIRE DUCHENNE Trial
Solid Biospace Gene Therapy SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile and has been generally well tolerated in 40 participants treated to […]