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A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy.

Primary* To compare the efficacy of RO7239361 (BMS-986089) to placebo in ambulatory boys with Duchenne Muscular Dystrophy.Secondary* To compare the efficacy of RO7239361 (BMS-986089) to placebo using the following tests:- 4 stair climb velocity (…

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo.

The objective of the study is to asses the efficacy and safety of intravenous ATB200 co-administration with oral AT2221 by evaluating the changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset…

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.