3 results
The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and femaleparticipants less than or equal to 6 months of age with IOPD.
The primary objective of the study is to evaluate the safety and tolerability of the Occlutech® AFR device by assessing the incidence of SADEs in the 3 months following implantation. Secondary objectives are related to safety and efficacy:• To…
The primary aim of the study is to compare the effect of PRO symptom monitoring with standard care on QoL both during and up to 1 year after treatment. Secondary aims are studying the effect of PRO symptom monitoring compared to standard care on the…