Clinical Trial: Natural History Study of Patients With MPS IIIA

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational




Official Title: An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Brief Summary: Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families.