A prospective natural history study for GM1 and GM2: 24-month data from the PRONTO study

Molecular Genetics and Metabolism 2026

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Authors: Roberto Giugliani, Andreas Hahn, Paul Harmatz, Bénédicte Héron, Aurélie Bourchany, Lara Cirnigliaro, Radhika Dhamija, Daniel A.D. Valle, Anna Ardissone, Spyros P. Batzios, Maurizio Scarpa, Aidan Gill
+2 more Christian Freitag, Laura López de Frutos
DOI: 10.1016/j.ymgme.2025.109430
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
109430–109430
Publication date
1 February 2026
Language
EN