Niemann-Pick Disease, Type C research hub

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

This study is looking at a new medicine called N-Acetyl-L-Leucine for Niemann-Pick Disease Type C. It compares the new medicine to a dummy treatment (placebo) to see if it's safe and helps people aged 4 and older with this condition.