Efficacy of arimoclomol combined with miglustat at months 3, 6, 9 and 12 of the double-blind, randomized, placebo-controlled NPC002 trial

Molecular Genetics and Metabolism 2026

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Authors: Eugen Mengel, Adrian A. Quartel, Christine í Dali

DOI: 10.1016/j.ymgme.2025.109548

Cited by: 0

Fields of Study & Subjects

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