SARA subgroup analysis based on disease duration in a phase 2 trial evaluating N-acetyl-L-leucine in GM2 gangliosidoses

Molecular Genetics and Metabolism 2026

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Authors: Marc C. Patterson, Janelle L. Raymond, Jorgji Kerthi, Bethany Zanrucha, Asante Hatcher, Taylor Fields, Ian Billington, Michael Strupp, Κyriakos Martakis, Κyriakos Martakis
DOI: 10.1016/j.ymgme.2025.109576
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109576–109576
Publication date
1 February 2026
Language
EN