not yet recruiting

Natural History Observational Study of MPS IIIa in SMC

key information

study id #: NCT04918641

condition: Sanfilippo Syndrome A

status: Not yet recruiting


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

intervention: assessment


last updated: February 26, 2022