Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2023
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
Epistemonikos ID: ba23204db43407441845c530a55259c93679b12b
First added on: May 15, 2024