MPS IIIA Disease Monitoring Program

Authors
Category Primary study
Registry of TrialsClinicalTrials.gov
Year 2026
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Epistemonikos ID: ce1af88f73ade7fd0d1801e11c06b67fc235effe
First added on: Oct 03, 2026