A two part seamless, open label, multi-center study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with type 1 spinal muscular atrophy.

drug protocol number
BP39056
drug sponsor
Roche
drug study
RO7034067
drug trial site
KFSH&RC-R
drug status
Ongoing
drug phase