Metabolic recovery with eculizumab precision therapy of CHAPLE disease
收藏NIAID Data Ecosystem2026-03-13 收录
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资源简介：
Complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE disease) is a lethal disease caused by genetic loss of the complement regulator CD55. We tested the complement C5 inhibitor eculizumab in a case series of 6 children. Over 14 months, fecal samples were collected for metagenomic sequencing of the gut microbiome.
创建时间：
2021-11-17
