A phase 1 dose-escalation study: Safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload

by Hugh Young Rienhoff, Vip Viprakasit, Lay Tay, Paul Harmatz, Elliott Vichinsky, Deborah Chirnomas, Janet L. Kwiatkowski, Amy Tapper, William Kramer, John B. Porter, Ellis J. Neufeld
Published 2018

Betibeglogene autotemcel gene therapy for non–β⁰/β⁰ genotype β-thalassemia

by Franco Locatelli, Alexis A. Thompson, Janet L. Kwiatkowski, John B. Porter, Adrian J. Thrasher, Suradej Hongeng, Martin G. Sauer, Isabelle Thuret, Ashutosh Lal, Mattia Algeri, Jennifer Schneiderman, Timothy S. Olson, Ben Carpenter, Persis J. Amrolia, Usanarat Anurathapan, Axel Schambach, Christian Chabannon, Manfred Schmidt, Ivan Labik, Heidi Elliot, Ruiting Guo, Mohammed Asmal, Richard A. Colvin, Mark C. Walters
Published 2022