N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

Niemann-Pick disease type C (NPC) is a rare autosomal recessive neurodegenerative lysosomal disease characterized by multiple symptoms such as progressive cerebellar ataxia and cognitive decline. The modified ...

Source: Trials - May 29, 2023 Category: General Medicine Authors: T Fields, T M. Bremova, I Billington, GC Churchill, W Evans, C Fields, A Galione, R Kay, T Mathieson, K Martakis, M Patterson, F Platt, M Factor and M Strupp Tags: Study protocol Source Type: research