Subdomain analysis of the 5-domain Niemann-Pick disease type C Clinical Severity Scale evaluating the long-term effects of N-acetyl-L-leucine in Niemann-Pick disease type C

Molecular Genetics and Metabolism 2026

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Authors: Marc C. Patterson, Jorgji Kerthi, Bethany Zanrucha, Janelle L. Raymond, Asante Hatcher, Taylor Fields, Ian Billington, Michael Strupp, Tatiana Brémovà-Ertl
DOI: 10.1016/j.ymgme.2025.109575
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Genetic Neurodegenerative Diseases

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109575–109575
Publication date
1 February 2026
Language
EN