Clinical Trial: A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
Study Status: COMPLETED
Recruit Status: COMPLETED
Condition: Mucopolysaccharidosis III-A
Study Type: OBSERVATIONAL
Official Title: A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA)
Brief Summary:
This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome).At least 10 participants (~50%) must be less than four years old at the time of the Parent/LAR consent.The study will have a screening process and 7 study visits, e.g. home visits, that will consist of serum collection and completion of a remote assessment of the Vineland Adaptive Behavior Scales 3rd Edition (Vineland-3)MPS IIIA remains a devastating disease…
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