Article

Economic Burden of Sanfilippo Syndrome in the United States

2023-11-01

Abstract excerpt

<title>Abstract</title> <p> <bold>Introduction:</bold> 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. <bold>Objectives:</bold> To develop a model to estimate the economic burden associated with Sanfilippo syndrome in the US using direct costs, indirect costs and...

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Literature Corpus work: 3d8c94f0-cb75-55b3-acc1-71f53e4f973f
DOI: 10.21203/rs.3.rs-3001450/v4

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