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
Primary Objective:To evaluate the efficacy of ELX/TEZ/IVA in subjects 6 through 11 years of age with CF, heterozygous for F508del and a MF mutation (F/MF)Secondary Objectives:• To evaluate the PD of ELX/TEZ/IVA• To evaluate the safety of ELX/TEZ/IVA