Quantitative longitudinal natural history of 8 gangliosidoses—conceptual framework and baseline data of the German 8-in-1 disease registry. A cross-sectional analysis-Reference-Cited by-同舟云学术

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Quantitative longitudinal natural history of 8 gangliosidoses—conceptual framework and baseline data of the German 8-in-1 disease registry. A cross-sectional analysis

Published:2022-12 Issue:12 Volume:24 Page:2434-2443
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

Ries Markus^ORCID,Mendoza Grecia,Arash-Kaps Laila,Amraoui Yasmina,Quack Folker,Hardt Brigitte,Diederich Stefan,Beck Michael,Mengel Eugen^ORCID

Publisher

Elsevier BV

Subject

Genetics (clinical)

Reference32 articles.

1. β-galactosidase deficiency (β-galactosidosis): GM1 gangliosidosis and Morquio B disease;Suzuki,2019

2. Gangliosides and gangliosidoses: principles of molecular and metabolic pathogenesis;Sandhoff;J Neurosci,2013

3. The GM2 gangliosidoses;Gravel,2019

4. Galactosialidosis;d’Azzo,2019

5. The GM2 gangliosidoses: unlocking the mysteries of pathogenesis and treatment;Toro;Neurosci Lett,2021

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Quantitative Systems Pharmacology for Rare Disease Drug Development;Journal of Pharmaceutical Sciences;2023-09

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