3 recruiting studies matching your filters
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard …
The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mann…
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandi…