5 results
To prevent avoidable visual impairment, in our controlled study, we want to contribute to the evidence of good treatment effects on visual functions (visual acuity both distances near and at distance, accuracy of accommodation), prevention of…
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.
Primary ObjectivePart1: To evaluate the safety and tolerability of intravenous (IV) administration of AL001 over up to 96 weeks in asymptomatic and symptomatic carriersof a granulin (GRN) mutation causative of frontotemporal dementia (FTD) and in…
The primary objective of this study is: Part 1: To evaluate the efficacy of AL001 compared with placebo as measured by CDR® plus NACC FTLD-SB.Part 2: To assess the long-term safety and tolerability of AL001 in participants who have completed 96…
Long-term safety and pharmacokinetics (PK) of neoGAA