Lemberg, Kathryn M. https://orcid.org/0000-0003-1511-9322
Gross, Andrea M.
Sproule, Lauren M.
Liewehr, David J.
Dombi, Eva
Baldwin, Andrea
Steinberg, Seth M.
Bornhorst, Miriam
Lodish, Maya
Blakeley, Jaishri O.
Widemann, Brigitte C.
Article History
Received: 15 February 2024
Revised: 19 July 2024
Accepted: 25 July 2024
First Online: 28 August 2024
Competing interests
: K.M.L is an inventor on the patent (WO2022046910) for prodrugs of 6-Mercaptopurine (not relevant to the current manuscript). M.B. has a paid advisory role on the external advisory board for the Koselugo Registry through Alexion (not relevant to this manuscript). J.O.B. is a co-investigator on clinical trials supported by Alexion, SpringWorks, and Takeda (not relevant to this manuscript) and serves as Executive Director for the Neurofibromatosis Therapeutic Acceleration Program within Johns Hopkins University. B.C.W. and A.M.G. have unpaid advisory roles for AstraZeneca and SpringWorks (not relevant to this manuscript).
: Informed consent was obtained from all participants on NCT00924196.