Learn more about our global Phase 3 HARBOR™ study for DM1 and our positive long-term MARINA-OLE™ data including vHOT (a measurement of myotonia) video and patient testimonials

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2021 FSHD Society IRC Annual Meeting

DUX4 siRNA Optimization for the Development of an Antibody Oligonucleotide Conjugate (AOC™) for the Treatment of FSHD