Individual participant outcomes after FLT201 AAV gene therapy for type 1 Gaucher disease: Two-year biomarker and clinical data

Molecular Genetics and Metabolism 2026

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Authors: Özlem Göker-Alpan, Pilar Giraldo, Reena Sharma, Ida Vanessa Döederlein Schwartz, Pamela Foulds, Philip Yin, Daniel Wolf, Simon Flynn

DOI: 10.1016/j.ymgme.2025.109434

Cited by: 0

Fields of Study & Subjects

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