Exemplifying a measurement validation strategy for rare- and ultra-rare diseases: measuring what matters in alpha-mannosidosis

Abstract Background Outcomes assessment in rare and ultra-rare diseases can be hampered by the paucity of condition-specific patient-reported outcome (PRO) measures that are known to be reliable, valid, and fit for purpose. It would be helpful to develop a strategy for measuring what matters to pati...

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Principais autores:	Carolyn E. Schwartz, Katrina Borowiec, Irene Koulinska, Heather Morgan, Bruce D. Rapkin
Formato:	Artigo
Idioma:	Inglês
Publicado em:	BMC 2026-02-01
coleção:	Orphanet Journal of Rare Diseases
Assuntos:	Measurement Patient-reported outcomes Proxy report Rare disease Ultra-rare disease Alpha-Mannosidosis
Acesso em linha:	https://doi.org/10.1186/s13023-026-04245-1
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