(MPS I)

Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, gives an overview of the treatment landscape for mucopolysaccharidosis type I.

There are two MPS I treatment options that are approved or recognized as efficacious – enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT). ERT involves intravenous iduronidase which is a life-long treatment usually given every week. Watch the video to learn more >>