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Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Crossref DOI link: https://doi.org/10.1186/s13023-021-01829-x

Published Online: 2021-05-12

Published Print: 2021-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Gissen, Paul https://orcid.org/0000-0002-9712-6122
Specchio, Nicola

Olaye, Andrew

Jain, Mohit

Butt, Thomas

Ghosh, Wrik

Ruban-Fell, Benjamin

Griffiths, Annabel

Camp, Charlotte

Sisic, Zlatko

Schwering, Christoph

Wibbeler, Eva

Trivisano, Marina

Lee, Laura

Nickel, Miriam

Mortensen, Amanda

Schulz, Angela
Funding

Funding for this research was provided by:

BioMarin
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Article History

Received: 9 October 2020

Accepted: 20 April 2021

First Online: 12 May 2021

Declarations

:

: No patient data was collected during this study.

: All the results presented in this article are in aggregate form, and no personally identifiable information was used for this study.

: PG, NS, CS, EW, MT, LL, MN, AS: no competing interests to declare; MJ, CC, TB: employees and shareholders of BioMarin; ZS, AO: employee and shareholder of BioMarin at time of study initiation; WG: employee of Costello Medical at time of study initiation; BRF, AG: employees of Costello Medical; AM: the BDFA receives funding from BioMarin but did not receive any funding associated with this study.

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