A prospective dose escalation study with ambroxol in patients with Sanflippo syndrome

Molecular Genetics and Metabolism 2026

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Authors: Özlem Göker-Alpan, Arooj Agha, Leah Marie Svarny, Margarita Ivanova
DOI: 10.1016/j.ymgme.2025.109433
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
109433–109433
Publication date
1 February 2026
Language
EN