PDF | Exemplifying a measurement validation strategy for rare- and ultra-rare diseases: measuring what matters in alpha-mannosidosis | ID: 6m3124487

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

Schwartz, Carolyn E.

Borowiec, Katrina

Koulinska, Irene

Morgan, Heather

Rapkin, Bruce D.

2026

Description

Keywords: Measurement, Patient-reported outcomes, Proxy report, Rare disease, Ultra-rare disease, Alpha-Mannosidosis, Lysosomal storage disorder, Caregivers

Springer Open.

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Citation

Carolyn E. Schwartz, Katrina Borowiec, Irene Koulinska, Heather Morgan, Bruce D. Rapkin. "Exemplifying a measurement validation strategy for rare- and ultra-rare diseases: measuring what matters in alpha-mannosidosis." Orphanet Journal of Rare Diseases, BioMed Central, London, v.21, no.1, 2026