Huub Jan Kleijn

What is the current knowledge on this topic? ○ Hereditary angioedema (HAE) is a rare disease characterized by recurrent episodes of severe tissue swelling. ○ Donidalorsen, an RNA-targeted antisense oligonucleotide that specifically reduces plasma prekallikrein production in the liver, significantly reduced HAE attack rates with an acceptable safety profile when administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) in clinical trials. What question did this study address?

Introduction Von Hippel–Lindau (VHL) disease is a hereditary syndrome transmitted in an autosomal dominant manner, in which the VHL protein is lost or functionally compromised due to germline mutation or deletion of the VHL gene.1, 2 Affected individuals are at risk of developing tumors in a number of organs including kidney, brain, and pancreas.

Supporting Information Filename Description psp413028-sup-0001-Supinfo1.pdfPDF document, 1.4 MB Supplementary file psp413028-sup-0002-Supinfo2.txtplain text document, 3 KB Supplementary file