The effect of the investigational gene therapy clemidsogene lanparvovec (RGX-121) on long-term levels of D2S6 in cerebrospinal fluid in patients with Hunter syndrome

Molecular Genetics and Metabolism 2026

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Authors: Paul Harmatz, Can H. Ficicioglu, Roberto Giugliani, Deepa Soundara Rajan, Debra-Lynn Day-Salvatore, Adam Gold, Robert A. Crozier, Leslie Magnus
DOI: 10.1016/j.ymgme.2025.109456
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Neurogenetic and Muscular Disorders Research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109456–109456
Publication date
1 February 2026
Language
EN