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Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Abstract:
Background: 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. Results: In this study, we have evaluated a simple metric, denoted annual severity increment score (ASIS), that measures ra... Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

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Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Pharmacology
Role:
Author
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Name:
Wellcome Trust
Funding agency for:
Platt, FM
Grant:
Investigator in Science (202834/Z/16/Z
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Name:
Royal Society
Funding agency for:
Platt, FM
Grant:
Investigator in Science (202834/Z/16/Z
More from this funder
Name:
Actelion
Funding agency for:
Vruchte, D
Grant:
SP4203
SP3775
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Name:
Niemann-Pick disease group UK
Funding agency for:
Vruchte, D
Grant:
SP4203
SP3775
More from this funder
Name:
Action Medical Research
Funding agency for:
Vruchte, D
Grant:
SP4203
SP3775
Publisher:
BioMed Central
Journal:
Orphanet Journal of Rare Diseases More from this journal
Volume:
13
Article number:
143
Publication date:
2018-08-16
Acceptance date:
2018-07-30
DOI:
ISSN:
1750-1172
Keywords:
Pubs id:
pubs:891715
UUID:
uuid:1ca607de-3abe-4334-8f1b-248d228b07be
Local pid:
pubs:891715
Deposit date:
2018-07-30

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