Based on regulatory agency feedback, the Company plans a registrational study that will evaluate hospitalization as the primary outcome measure, supported by multiple functional assessments as secondary outcome measures.
LONDON, July 6, 2026 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, today announced agreement with regulatory agencies in the U.S., U.K. and Canada on design for a registrational clinical study intended to assess the safety and efficacy of the Company's investigational therapy AMO-02 (oral tideglusib) in the treatment of congenital myotonic dystrophy type 1 (cDM1).
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