A Phase 1/2 first-in-human study to evaluate the safety, tolerability, and pharmacokinetics of UX053 in patients with glycogen storage disease type III

UX053 - Ultragenyx, Arcturus Therap

https://www.sciencedirect.com/science/article/pii/S1096719224001355

David F. Rodriguez-Buritica 1; Tahseen Mozaffar 2; Giacomo P. Comi 3; Montserrat Morales-Conejo 4; Han C. Phan 5; Syeda Rahman 6; Diana Luca 6; Tim Chiu 6; Manju Gupta 6; Rachael Hawtin 6; Andrew Grimm 6

Jan 13, 2024