Clinical DevelopmentDYNE-101 delivers consistent/sustained treatment benefit at 12mo, showing improvement in myotonia, muscle strength, mobility, and patient-reported outcomes, with consistent safety.
Competitive PositioningVHOT strengthens DYNE-101's competitive positioning—competitor might be powered for multiple endpoints.
Regulatory ApprovalDyne announced FDA Breakthrough Therapy Designation and updated plan for accelerated approval in myotonic dystrophy type 1.