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Sanfilippo syndrome: consensus guidelines for clinical care

27 Oct 2022

Abstract excerpt

Sanfilippo syndrome is a group of rare, complex, and progressive neurodegenerative lysosomal storage disorders that is characterized by childhood dementia. The clinical management of patients with progressive neurological decline and multisystem involvement requires a multidisciplinary team with experience in the management of neurodegenerative disorders. Best practice guidelines for the clinical management of...

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PMID: 36303195
DOI: 10.1186/s13023-022-02484-6

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