Lyso-Gb1 dynamics as a surrogate biomarker in type 1 Gaucher disease treated with FLT201 AAV gene therapy

Molecular Genetics and Metabolism 2026

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Authors: Daniel Wolf, Reena Sharma, Pilar Giraldo, Ida Vanessa Döederlein Schwartz, Özlem Göker-Alpan, Pamela Foulds, Philip Yin, Simon Flynn, Derralynn A. Hughes
DOI: 10.1016/j.ymgme.2025.109677
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Virus-based gene therapy research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109677–109677
Publication date
1 February 2026
Language
EN