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A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy

Primary objective:Evaluate the safety and tolerability of single ascending doses of WVE-210201 in patients with DMD. Secondary objective:Assess the pharmacokinetics (PK) of WVE-210201 in patients with DMD.

FSHD Longitudinal Biomarker Preparatory Study

The primary objective of this study is the further evaluation and optimization of skeletal muscle tissue and MRI biomarkers for a planned POC study of FTX-1821 in FSHD. Therefore, this study is not a drug trial but rather a preparatory biomarker…