five

Metabolic recovery with eculizumab precision therapy of CHAPLE disease

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https://www.ncbi.nlm.nih.gov/sra/SRP259219
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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
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