A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease

• Main Authors: Diqi Zhu (Author), Jiacong Zhu (Author), Wenjuan Qiu (Author), Benzhen Wang (Author), Lin Liu (Author), Xiaodan Yu (Author), Zhenheng Ou (Author), Guangsong Shan (Author), Jian Wang (Author), Bin Li (Author), Xiaokang Chen (Author), Cong Liu (Author), Zipu Li (Author), Lijun Fu (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: A high prevalence of infantile-onset Pompe disease (IOPD) in the Chinese population has been noted, but there are currently no reported clinical trials of enzyme replacement therapy (ERT) for IOPD in this population. The purpose of this study was to evaluate the efficacy and safety of al...