Indication
- For the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
Last updated on 12/9/2020Document Title | Year | Source |
A two part seamless, open-label, multicenter study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy. | 2020 | Hoffmann-La Roche |
A two part seamless, multi-center randomized, placebo-controlled, double-blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of risdiplam (RO7034067) in type 2 and 3 spinal muscular atrophy patients. | 2020 | Hoffmann-La Roche |