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 WMORecruiting
The primary objective of Arm 1 is to evaluate the overall response rate (ORR) as determined by an independent radiology review committee (IRC) and measured by the proportion of patients with best overall confirmed response of complete response (CR)…