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REGENXBIO Shares Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202
REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of
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Solid Biosciences Shares Interim Positive Update on Phase 1/2 INSPIRE DUCHENNE Trial
Solid Biosciences Inc. has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE
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ITF Therapeutics Shares Community Letter and Presents New Data and Analyses on DUVYZAT® (givinostat)
ITF shared a community letter and presented new data, including long-term safety observations based on data
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Solid Biosciences Provides Positive Interim Clinical Update from Phase 1/2 INSPIRE DUCHENNE Trial
SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile
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Guest Voice: Navigating the windy road of rare disease specialists
Living with a rare disease is incredibly complex, with countless layers to navigate. Managing daily
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Capricor Therapeutics Announces New PDUFA Date for Deramiocel
Capricor Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has lifted the
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MDA 2026: Keynote speaker to MDA community: ‘Your voice is powerful’
At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are
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Capricor announces the FDA to review deramiocel for Duchenne by August 2026
As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA
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Edgewise Therapeutics, a CureDuchenne funded company, announces positive long-term Sevasemten data in Becker muscular dystrophy patients.
Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained
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Edgewise Announces Positive Long-Term Sevasemten Data
Edgewise Therapeutics, Inc. has shared long-term data from its MESA open-label extension study of sevasemten
