Clinical features and enzyme replacement therapy in 10 children with Fabry disease

• Main Authors: Qian Li (Author), Jing Wang (Author), Minle Tian (Author), Zhenle Yang (Author), Lichun Yu (Author), Suwen Liu (Author), Cong Wang (Author), Xiaoyuan Wang (Author), Shuzhen Sun (Author)
• Format: Book
• Published: Frontiers Media S.A., 2023-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

ObjectiveTo summarize the clinical features, diagnosis and enzyme replacement therapy(ERT) of Fabry disease (FD) in children.MethodsThe clinical data, laboratory tests, genetic variations and treatment of 10 FD children diagnosed in Shandong Provincial Hospital from September 2020 to June 2022 were...