Economic burden of Sanfilippo syndrome in the United States
Sanfilippo syndrome is a rare lysosomal storage disease, however the severe burden associated with the disease for individual families suggests a possible cumulative impact. Our model represents the first disease burden value estimate associated with Sanfilippo syndrome, and underscores the substantial morbidity and mortality burden of Sanfilippo syndrome.
Authors
- Mikael Svensson (ORCID: https://orcid.org/0000-0003-1113-7478)
- Frederick Ashby (ORCID: https://orcid.org/0000-0002-6975-8990)
- Coy D. Heldermon (ORCID: https://orcid.org/0000-0002-2148-2868)
- Haesuk Park (ORCID: https://orcid.org/0000-0003-3299-8111)
Institutions
- University of Florida (US)
- Florida College (US)
Publication Details
- Journal
- Archives of Public Health
- Published
- 2025-11-10
- DOI
- https://doi.org/10.1186/s13690-025-01733-x
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National MPS Society
- National Institutes of Health
- National Institute of Neurological Disorders and Stroke