A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative
A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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Contents
Gaucher disease (GD) is a rare autosomal recessive lysosomal storage disorder. GD types 2 and 3 are known as neuronopathic Gaucher disease (nGD) because they have brain involvement that progresses over time. Implementing a systematic approach to the collection of real-world clinical and patient-relevant outcomes data in nGD presents an opportunity...
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Full title
A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative
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TN_cdi_doaj_primary_oai_doaj_org_article_966c96d854524e36b98f9a6d422e8753
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_966c96d854524e36b98f9a6d422e8753
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-023-02828-w
