Presymptomatic and symptomatic disease stage-dependent outcomes of cerliponase alfa treatment in siblings with CLN2 disease

Molecular Genetics and Metabolism 2026

View at publisher
Authors: Lena Marie Westermann, Lara Y. Ruhberg, Susanne Lezius, Luca Hagenah, Eva Heemann, Miriam Nickel, Christoph Schwering, Christian Posern, Angela Schulz
DOI: 10.1016/j.ymgme.2025.109668
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Metabolism and Genetic Disorders

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109668–109668
Publication date
1 February 2026
Language
EN