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

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 differ...

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Bibliographic Details
Main Authors: Mario Cortina-Borja, Danielle te Vruchte, Eugen Mengel, Yasmin Amraoui, Jackie Imrie, Simon A. Jones, Christine i Dali, Paul Fineran, Thomas Kirkegaard, Heiko Runz, Robin Lachmann, Tatiana Bremova-Ertl, Michael Strupp, Frances M. Platt
Format: Article
Language:English
Published: BMC 2018-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Niemann-Pick disease type C
NPC
Annual severity increment score
ASIS
Clinical severity scale
Clinical trials
Online Access:http://link.springer.com/article/10.1186/s13023-018-0880-9

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http://link.springer.com/article/10.1186/s13023-018-0880-9

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