Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Sp
Brief Summary: Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic, pharmacodynamics, and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate RO7034067 for 24-months at the dose selected in Part 1.