Natural History Observational Study of MPS IIIa in SMC

Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.

Inclusion Criteria:

• Confirmed diagnosis of MPS IIIA
• Has the ability to comply with protocol requirements, in the opinion of the investigator

Exclusion Criteria:

• Patient's assent is unattainable, or the patient's parent(s), or patient's legally authorized representative(s) is/are unable to understand the nature, scope, and possible consequences of the study, or do/does not agree to comply with the protocol defined schedule of assessments.