Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments
Orphanet J Rare Dis. 2024 Aug 8;19(1):290. doi: 10.1186/s13023-024-03277-9. ABSTRACT BACKGROUND: CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is …