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Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

Published Online: 2021-02-12

Published Print: 2021-12

Authors

Patterson, Marc C.

Lloyd-Price, Lucy

Guldberg, Christina

Doll, Helen

Burbridge, Claire

Chladek, Michael

íDali, Christine

Mengel, Eugen

Symonds, Tara
Clinical Trials ^BETA

Clinical trials referenced in this document:

nct02612129 at ClinicalTrials.gov

Documents that mention this clinical trial

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
https://doi.org/10.1186/s13023-021-01719-2
Funding

Funding for this research was provided by:

Orphazyme A/S
License Information

Text and Data Mining valid from 2021-02-12

Version of Record valid from 2021-02-12
More Information

Article History

Received: 26 October 2020

Accepted: 1 February 2021

First Online: 12 February 2021

Ethics approval and consent to participate

: All study procedures were in accordance with ethical standards of the 1964 Helsinki Declaration and its later amendments, relevant laws, institutional guidelines, and approved by an institutional review board. Participant informed consent (written or verbal) was obtained as approved by the institutional review board.

: Not applicable.

: CG and CD are employees of Orphazyme A/S. TS, MP, LLP, HD, CB, MC, and EM declare no competing interests.

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