International Niemann-Pick Disease Registry (INPDR): Enrollment characteristics of patients enrolled in the largest database of patients with ASMD

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Authors: Roberto Giugliani, Jenny L. Carwile, Eugen Mengel, Shaun Bolton, Justin Hopkin, Maja Gašparić, Pablo Manuel Bianculli, Michal Inbar‐Feigenberg, Reena Sharma, Tarekegn Geberhiwot
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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
109429–109429
Publication date
1 February 2026
Language
EN