3 results
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…
Primary: efficacy, assessed by best-corrected visual acuity and SD-OCT of 1 eye (*study eye*). Secondary: retina anatomy of the study eye, safety and tolerability, PK, PD.Exploratory: Best-corrected visual acuity, SD-OCT and retina anatomy of the…
The objectives of the study are to evaluate the following in patients with a documented history of EPP:•determine whether CUV1647 can reduce the number of phototoxic reactions in patients with EPP•determine whether CUV1647 can reduce the severity of…