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JLK Sanfilippo Research Foundation

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Homepage: http://www.jlksanfilippofoundation.com/

Recent papers from JLK Sanfilippo Research Foundation

Cerebrospinal fluid Heparan sulfate as a biomarker for neuronopathic mucopolysaccharidoses: Rationale and regulatory challengesMolecular Genetics and Metabolism · 2026
AMCP Market Insights: The role of managed care in improving patient care in mucopolysaccharidosesJournal of Managed Care & Specialty Pharmacy · 2026
Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic MucopolysaccharidosesMolecular Genetics and Metabolism · 2024
Sanfilippo Syndrome: The Tale of a Challenging DiagnosisJournal of Inborn Errors of Metabolism and Screening · 2020