Real world data of patients with lysosomal acid lipase deficiency under enzyme replacement therapy experience from a reference center in São Paulo, Brazil

Molecular Genetics and Metabolism 2026

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Authors: Carolina de Souza Araujo, Carmen Silvia Curiati Mendes, Marco A. Curiati, Sandra Obikawa Kyosen, Guilherme Sotto Battiston, Fernanda Araujo Romera, Maria Isabel Riesemberg, Andre Luis Ferreira, Ana Margarida Martins
DOI: 10.1016/j.ymgme.2025.109313
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109313–109313
Publication date
1 February 2026
Language
EN