This is a multicenter, open-label study to study the safety and characteristics of DNL310, an investigational drug designed to treat both the body and brain manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). The plan is for each participant to start at a low dose and increase over time until a final dose is achieved. Blood, urine and CSF samples will be taken, and tests and assessments will be done to see how your child is doing in the study.
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
20-1303