Patient-reported outcomes in MPS I patients treating with lepunafusp alfa: 3 year experience

Molecular Genetics and Metabolism 2026

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Authors: Marco A. Curiati, Felippe R.E.O. Previdi, Jessica Grasiela Araujo Espolaor, Edna Tiemi Sakata, Marcelo Hideki Yamamoto, Carmen Silvia Curiati Mendes, Ana Margarida Martins
DOI: 10.1016/j.ymgme.2025.109376
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Hemophilia Treatment and Research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109376–109376
Publication date
1 February 2026
Language
EN