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Intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II: Final report of 5-year results from a Japanese open-label phase 1/2 study

Joo-Hyun Seo, Motomichi Kosuga, Takashi Hamazaki, Haruo Shintaku, Torayuki Okuyama

2023Molecular Genetics and Metabolism20 citationsDOI

Topics & Concepts

Enzyme replacement therapyMedicineAdverse effectHunter syndromeMucopolysaccharidosis type IIInternal medicineInterim analysisCerebrospinal fluidMucopolysaccharidosis type IGastroenterologyClinical trialDiseaseLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusIntestinal Malrotation and Obstruction Disorders
Intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II: Final report of 5-year results from a Japanese open-label phase 1/2 study | Litcius