Effect of clemidsogene lanparvovec (RGX-121), an investigational gene therapy, on neurodevelopmental outcomes in patients with Hunter syndrome

Molecular Genetics and Metabolism 2026

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Authors: Can H. Ficicioglu, Roberto Giugliani, Paul Harmatz, Deepa Soundara Rajan, Debra-Lynn Day-Salvatore, Adam Gold, Robert A. Crozier, Leslie Magnus
DOI: 10.1016/j.ymgme.2025.109413
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Genetics and Neurodevelopmental Disorders

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109413–109413
Publication date
1 February 2026
Language
EN