Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and
Brief Summary: Multi-center, randomized, double-blind, placebo-controlled, Phase II study to assess the safety, tolerability, pharmacokinetic, pharmacodynamics, and efficacy of RO7034067 in adult and pediatric patients with Type 2 and Type 3 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) of RO7034067 for 12-weeks and a confirmatory part (Part 2) of RO7034067 for 24-months.