Sanfilippo Syndrome Type A Clinical Trial
Official title:
A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.
n/a
Status | Clinical Trial | Phase | |
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Completed |
NCT01873911 -
Neurobehavioral Phenotypes in MPS III
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