Article

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

2022-10-20

Abstract excerpt

<title>Abstract</title> <p>Background 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 amino acid N-acetyl-leucine has been associated with positive symptomatic and neuroprotective, disease-modifying effects in various studies, including animal models of...

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Literature Corpus work: d756ba9b-6f18-5d27-9d87-e5dd261eb097
DOI: 10.21203/rs.3.rs-2048685/v1

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