"Hunter syndrome is an X-linked recessive disorder caused by mutations in the iduronate 2-sulfatase (IDS) gene, responsible for the degradation of heparan and dermatan sulfate. Mutations of IDS lead to accumulation of proteoglycan products in tissues, leading to organ dysfunction and growth abnormalities 3."
"The mainstay of treatment for mucopolysaccharidoses is enzyme replacement therapy (ERT). For Hunter syndrome, this consists of replacement with idursulfase or tividenofusp alfa-eknm administered via intravenous infusions. Enzyme replacement therapy in Hunter syndrome has been shown to produce symptomatic improvement in somatic symptoms and appears to slow disease progression 6. However, because idursulfase does not cross the blood-brain barrier, it does not affect cognitive decline 7; tividenofusp alfa-eknm does cross the blood-brain barrier but its clinical benefits are not yet certain (c.2026). Haematopoietic cell transplantation has been shown to improve clinical outcomes but also does not prevent cognitive decline 8,9."
"The mainstay of treatment for mucopolysaccharidoses is enzyme replacement therapy (ERT). For Hunter syndrome, this consists of replacement with idursulfase or tividenofusp alfa-eknm administered via intravenous infusions. Enzyme replacement therapy in Hunter syndrome has been shown to produce symptomatic improvement in somatic symptoms and appears to slow disease progression 6. However, because idursulfase does not cross the blood-brain barrier, it does not affect cognitive decline 7; tividenofusp alfa-eknm does cross the blood-brain barrier but its clinical benefits are not yet certain (c.2026). Haematopoietic cell transplantation has been shown to improve clinical outcomes but also does not prevent cognitive decline 8,9."
Expected headings
"MRI"
"The mainstay of treatment for mucopolysaccharidoses is enzyme replacement therapy (ERT). For Hunter syndrome, this consists of replacement with idursulfase or tividenofusp alfa-eknm administered via intravenous infusions. Enzyme replacement therapy in Hunter syndrome has been shown to produce symptomatic improvement in somatic symptoms and appears to slow disease progression 6. However, because idursulfase does not cross the blood-brain barrier, it does not affect cognitive decline 7; tividenofusp alfa-eknm does cross the blood-brain barrier but its clinical benefits are not yet certain (c.2026). Haematopoietic cell transplantation has been shown to improve clinical outcomes but also does not prevent cognitive decline 8,9."