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

David Rodriguez‐Buritica(The University of Texas Health Science Center at Houston), Andrew Grimm(Ultragenyx Pharmaceutical (United States)), Rachael E. Hawtin(Ultragenyx Pharmaceutical (United States)), Syeda Rahman(Ultragenyx Pharmaceutical (United States)), Montserrat Morales‐Conejo(Hospital Universitario 12 De Octubre), Tahseen Mozaffar(University of North Carolina at Chapel Hill), Manju Gupta(Ultragenyx Pharmaceutical (United States)), H. Phan(Rare Disease Therapeutics (United States)), Tim Chiu(Hospital for Sick Children), Diana Luca(Ultragenyx Pharmaceutical (United States)), Giacomo P. Comi(University of Milan)
Molecular Genetics and Metabolism
April 1, 2024
10.1016/j.ymgme.2024.108250
Cited by 0

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