Article
Population Pharmacokinetic-Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration.
Nucleic acid therapeutics - 1 Jun 2020
Goel Varun, Gosselin Nathalie H, Jomphe Claudia, Zhang Xiaoping, Marier Jean-Francois, Robbie Gabriel J
Abstract excerpt
Hereditary transthyretin-mediated amyloidosis is an inherited, rapidly progressive, life-threatening disease caused by mutated transthyretin (TTR) protein. Patisiran is a small interfering RNA (siRNA) formulated in a lipid nanoparticle that inhibits hepatic TTR protein synthesis by RNA interference. We have developed an indirect-response pharmacokinetic-pharmacodynamic model relating plasma siRNA (ALN-18328)...
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