Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C...
Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials
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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
Niemann-Pick disease type C (NPC) is a lysosomal storage disease with a heterogeneous neurodegenerative clinical course. Multiple therapies are in clinical trials and inclusion criteria are currently mainly based on age and neurological signs, not taking into consideration differential individual rates of disease progression.
In this study, we h...
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Full title
Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials
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TN_cdi_doaj_primary_oai_doaj_org_article_5ac3fe3f7d0c47d295617be6933c0b47
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_5ac3fe3f7d0c47d295617be6933c0b47
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-018-0880-9
