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GM1 gangliosidosis type 1

Infantile GM1 gangliosidosisGangliosidosis Generalized GM1 Type 1
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Review Articles from PubMed

Review articles summarize what is currently known about a disease. They discuss research previously published by others. The terms "GM1 gangliosidosis type 1" returned 0 free, full-text review articles. First 0 results:
The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis.
Last Updated: Nov 23, 2020

Type 1 GM1 gangliosidosis is an ultra-rare, rapidly fatal lysosomal storage disorder, with life expectancy of <3 years of age. To date, only one prospective natural history study of limited size has been reported. Thus, there is a need for additional research to provide a better ...

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Editorials from PubMed

Editorial articles describe the outcome of a single study. They are the published results of original research. The terms "GM1 gangliosidosis type 1" returned 0 free, full-text editorial articles. First 0 results:
No publications were found for this category.
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Research Articles from PubMed

Research articles describe the outcome of a single study. They are the published results of original research. The terms "GM1 gangliosidosis type 1" returned 9 free, full-text research articles. First few results:
A new type of pharmacological chaperone for G-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.
Last Updated: Nov 25, 2017

N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for G-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type ...

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9 Free Research Articles 17 Research Articles