Economic burden of Sanfilippo syndrome in the United States.

ABSTRACT:

Introduction

Sanfilippo syndrome is a rare disease and fatal genetic disorder with no FDA-approved treatment in the United States (US), and no comprehensive assessment of economic disease burden is available.

Objectives

To develop a model to project the economic burden associated with Sanfilippo syndrome in the US using direct costs, indirect costs and valued intangibles (disability-adjusted life years, or DALYs) from 2023 onward.

Design and setting

A multistage comorbidity model was generated based on Sanfilippo syndrome symptoms, and disability weights from the 2010 Global Burden of Disease Study. Attributable increase in caregiver mental health burden was estimated using data from the CDC National Comorbidity Survey and retrospective studies on caregiver burden. Di

SUBMITTER: Ashby F 

PROVIDER: S-EPMC12604229 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature