Lv11
60 积分 2024-08-13 加入
An expert consensus on the recommendations for the use of biomarkers in Fabry disease
3小时前
待确认
抗肿瘤新药突破性治疗药物认定的标准及考虑
12天前
已完结
Breakthrough therapy designations in China and the United States
12天前
已完结
Assessment of the breakthrough-therapy-designated drugs granted in China: A pooled analysis 2020–2022
12天前
已完结
突破性治疗药物程序在药品注册体系中的作用及展望
13天前
已完结
The emerging phenotype of late-onset Pompe disease: A systematic literature review
1个月前
已完结
Pompe Disease in Infants: Improving the Prognosis by Newborn Screening and Early Treatment
1个月前
已完结
Mini-COMET study: Safety and efficacy data after avalglucosidase alfa dosing for ≥ 145 weeks in participants with infantile-onset Pompe disease (IOPD) who had demonstrated clinical decline or sub-optimal response while receiving alglucosidase alfa
1个月前
已完结
Baby-COMET: Safety of avalglucosidase alfa after repeat dosing in treatment-naïve participants with infantile-onset Pompe disease (IOPD)
1个月前
已完结
Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry
1个月前
已关闭
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