Impact of ERT and follow-up of 17 patients from the same family with a mild form of MPS II

Bruno de Oliveira Stephan,Caio Robledo Quaio,Gustavo Marquezani Spolador,Ana Carolina de Paula,Marco Antônio Curiati,Ana Maria Martins,Gabriela Nunes Leal,Artur Tenorio, Simone Finzi,Flavia Teixeira Chimelo,Carla Gentile Matas,Rachel Sayuri Honjo,Debora Romeo Bertola,Chong Ae Kim

Clinics（2022）

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摘要

•Clinical variability within a large family with a slowly progressive form of MPS II.•Despite being safe, ERT with α or β-Idursulfase might not prevent disease progression.

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