2 results
Not approvedWill not start
Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…
Approved WMOCompleted
The study will be performed in 2 parts, Parts 1 and 2. The volunteers can only participate in one of the two parts of this study. The purpose of Part 1 of the study is to investigate to what extent RO6806127 is tolerated. It will also be…