This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy.~This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.