3 results
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…
All efficacy study objectives will be assessed in the pediatric AS study population of subjects who are 4 to 12 years old. All safety study objectives will be assessed in the pediatric AS study population of subjects who are 2 to 12 years old.…
Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI for the total group (both prenatal and postnatal diagnosis) and the prenatal diagnosis group, which is needed to…