Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
study id #: NCT04088734
condition: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
status: Recruiting
purpose:Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
intervention: ABO-102
results: https://clinicaltrials.gov/ct2/show/results/NCT04088734
last updated: February 26, 2022
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