Long-term cardiac outcomes in attenuated MPS I patients under enzyme replacement therapy: A retrospective study from paediatric to adult metabolic centres

Molecular Genetics and Metabolism 2026

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Authors: Daniela Castillo Garcia, Meera V. Patel, Manuela Aimufua, Peter I. Woolfson, Reena Sharma, Ana Jovanović, Janet Gorton, Mairead A. Jones, Laura J. Bell, Daniel Hand, Maria De Castro Lopez, Eduardo Padilla
+4 more Laura Kusyk, Karen L. Tylee, Simon Allan Jones, Karolina M. Stępień
DOI: 10.1016/j.ymgme.2025.109358
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
109358–109358
Publication date
1 February 2026
Language
EN