Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

BACKGROUND: Disease-specific patient-reported outcome measures (PROMs) are fundamental to understanding the impact on, and expectations of, patients with genetic disorders, and can facilitate constructive and educated conversations about treatments and outcomes. However, generic PROMs may fail to ca...

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Detalles Bibliográficos
Autores principales: Elstein, Deborah, Belmatoug, Nadia, Deegan, Patrick, Göker-Alpan, Özlem, Hughes, Derralynn A., Schwartz, Ida Vanessa D., Weinreb, Neal, Bonner, Nicola, Panter, Charlotte, Fountain, Donna, Lenny, Andrew, Longworth, Louise, Miller, Rachael, Shah, Koonal, Schenk, Jörn, Sen, Rohini, Zimran, Ari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8734239/
https://www.ncbi.nlm.nih.gov/pubmed/34991656
http://dx.doi.org/10.1186/s13023-021-02163-y

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