Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) (S23.006)

Nicholas Johnson,John Day,Johanna Hamel,Jeffrey Statland, S.H. Subramony,W. David Arnold,Charles Thornton,Matthew Wicklund,Payam Soltanzadeh, Ben Knisley, Varun Goel,Kelly DiTrapani, Chao-Yin Chen, Kristi R. Clark, Alissa Peters, Carrie Heusner,Husam Younis, Mohita Kumar,Li-Jung Tai,Elizabeth Ackermann

Neurology（2022）

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摘要

The primary objective of the MARINA study is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 in adults with DM1.

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