7 recruiting studies matching your filters
This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick …
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (…
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 …
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick…
We are trying to identify factors associated with improved quality of life and fewer PD symptoms. We are attempting to identify practices, beliefs, and therapi…
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…