The purpose of this study is to compare the safety and efficacy of an investigational product, DNL310, to the current standard of care therapy, Idursulfase, in children with MPS II. The study will be randomized and double-blinded, which means participants and the study team will not know which drug is being given.
Thank you for your interest, but this study is recruiting by invitation only.
United States (Nationwide)
Joseph Muenzer
Pediatrics - Genetics and Metabolism
Clinical or Medical
Interventional
Genetics and Genetic Disorders
Rare Diseases
22-0709
Copyright © 2013-2022 The NC TraCS Institute, the integrated home of the NIH Clinical and Translational Science Awards (CTSA) Program at UNC-CH. This website is made possible by CTSA Grant UL1TR002489 and the National Center for Advancing Translational Sciences.
Questions?
