Psychometric Validation of the CLN2 Quality of Life Questionnaire in Participants with CLN2 Disease Treated with Cerliponase Alfa
This study is the first to validate the CLN2 QoL and to estimate the CID of this instrument in CLN2 patients. Our results show good validity and reliability of this tool.
Authors
- Thomas Butt (ORCID: https://orcid.org/0000-0002-0387-4550)
- Jennifer Quinn (ORCID: https://orcid.org/0000-0002-2428-7662)
- Emily de los Reyes (ORCID: https://orcid.org/0000-0002-1123-6903)
- Nicola Specchio (ORCID: https://orcid.org/0000-0002-8120-0287)
- Clemence Due (ORCID: https://orcid.org/0000-0001-6485-6076)
- Paul Gissen (ORCID: https://orcid.org/0000-0002-9712-6122)
- Angela Schulz (ORCID: https://orcid.org/0000-0001-9156-4861)
Institutions
- Nationwide Children's Hospital (US)
- Universität Hamburg (DE)
- Great Ormond Street Hospital (GB)
- National Institute for Health and Care Research (GB)
- Debiopharm (Switzerland) (CH)
- BioMarin (United Kingdom) (GB)
- University Medical Center Hamburg-Eppendorf (DE)
- Bambino Gesù Children's Hospital (IT)
- University College London (GB)
- KU Leuven (BE)
Publication Details
- Journal
- Healthcare
- Published
- 2024-11-08
- DOI
- https://doi.org/10.3390/healthcare12222229
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.33
Funders
- BioMarin Pharmaceutical