International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in...
International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study
About this item
Full title
Author / Creator
Evans, William , Patterson, Marc , Platt, Frances , Guldberg, Christina , Mathieson, Toni , Pacey, Jessica , Berry-Kravis, Elizabeth , Farhat, Nicole , Gascon, Jordi , Geberhiwot, Tarek , Gissen, Paul , Giugliani, Roberto , Hastings, Caroline , Héron, Bénédicte , Imrie, Jackie , Jones, Simon , Lachmann, Robin , Mengel, Eugen , Pineda, Mercedes , Porter, Denny , Runz, Heiko , Stampfer, Miriam , Strupp, Michael , Walterfang, Mark , the Core Working Group for the Delphi Study and Core Working Group for the Delphi Study
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
Several scales have been developed in the past two decades to evaluate Niemann-Pick disease Type C (NPC) severity in clinical practice and trials. However, a lack of clarity concerning which scale to use in each setting is preventing the use of standardised assessments across the world, resulting in incomparable data sets and clinical trial outcome...
Alternative Titles
Full title
International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study
Authors, Artists and Contributors
Author / Creator
Patterson, Marc
Platt, Frances
Guldberg, Christina
Mathieson, Toni
Pacey, Jessica
Berry-Kravis, Elizabeth
Farhat, Nicole
Gascon, Jordi
Geberhiwot, Tarek
Gissen, Paul
Giugliani, Roberto
Hastings, Caroline
Héron, Bénédicte
Imrie, Jackie
Jones, Simon
Lachmann, Robin
Mengel, Eugen
Pineda, Mercedes
Porter, Denny
Runz, Heiko
Stampfer, Miriam
Strupp, Michael
Walterfang, Mark
the Core Working Group for the Delphi Study
Core Working Group for the Delphi Study
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_46cfd0e5c0d2451ea0832be576c10057
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_46cfd0e5c0d2451ea0832be576c10057
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-021-02115-6
