Natural History Observational Study of MPS IIIa in SMC
study id #: NCT04918641
condition: Sanfilippo Syndrome A
status: Not yet recruiting
purpose:To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.
intervention: assessment
results: https://clinicaltrials.gov/ct2/show/results/NCT04918641
last updated: February 26, 2022
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