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Zhengqing Qiu

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Open access Oct 2026

Safety, Efficacy, and Pharmacokinetics of Agalsidase Alfa in Treatment-Naive Chinese Patients with Fabry Disease: 52-Week Phase 3, Open-Label, Single-Arm Study.

INTRODUCTION Clinical data on the efficacy, tolerability, and pharmacokinetics of agalsidase alfa in Chinese patients with Fabry disease are limited. We evaluated the safety, efficacy, and pharmacokinetics of agalsidase alfa in Chinese patients who are treatment-naive over 52 weeks. METHODS This was an open-label, no...

Hong Ren, Jian-Hua Mao, Rong Wang et al. · 0 citations
Open access Aug 2026

Improving molecular diagnosis of fabry disease: functional validation of novel splicing variants in GLA

The findings highlight the critical role of robust functional splicing assays in interpreting variant pathogenicity, establishing accurate genotype-phenotype correlations, and ultimately facilitating precision medicine for FD.

Jingwen Zhou, Ai-Ping Mao, Jie Feng et al. · 0 citations

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