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3 resultsUpdated October 24, 2026

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1

Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains

Orphanet Journal of Rare Diseases 19(1) (2024)

Emily McDool; Philip A. Powell; Jill Carlton

2

Humanistic burden of mucopolysaccharidoses: a systematic literature review

Current Medical Research and Opinion 40(4) (2024)

Joyce Wai-Yin Li; Kevin Yan; Chakrapani Balijepalli; Eric Druyts

3

Caregiver Reports of Neurodevelopmental Functions in Pediatric Lysosomal Storage Disorders: A Scoping Review

Journal of Inherited Metabolic Disease 49(3) (2026)

Eileen Gillan; Beth Leiro; Amanda M. Nagy; Florian Eichler; Elise L. Townsend