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 WMOPending
The primary objective of this study is to assess the effectiveness and cost-effectiveness in terms of the number of surgeries prevented of a combination therapy versus usual care.The secondary objective of this study is to assess the effectiveness…