The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis.

Frederick M Lang,Paul Korner,Mark Harnett,Ajith Karunakara,Cynthia J Tifft

Molecular Genetics and Metabolism（2020）

引用 16|浏览3

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摘要

This study highlights the significant burden of illness in this disease and provides critical natural history data to drive earlier diagnosis, inform clinical trial design, and facilitate family counseling.

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关键词

GM1 gangliosidosis,Type 1,Natural history study,Lysosomal storage disorder,Meta-analysis,Literature review

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