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The Journal of Clinical Investigation|June 12, 2018
Dominant-negative IKZF1 mutations cause a T, B, and myeloid cell combined immunodeficiencyDavid Boutboul, Hye Sun Kuehn, Zoé Van de Wyngaert, et al.JAMA Oncology|September 16, 2021
Carfilzomib, Lenalidomide, and Dexamethasone Followed by Lenalidomide Maintenance for Prevention of Symptomatic Multiple Myeloma in Patients With High-risk Smoldering Myeloma: A Phase 2 Nonrandomized Controlled TrialDickran Kazandjian, Elizabeth Hill, Alexander Dew, et al.The New England Journal of Medicine|March 17, 2016
Loss of B Cells in Patients with Heterozygous Mutations in IKAROSH S Kuehn, B Boisson, C Cunningham-Rundles, et al.Blood|April 21, 2023
Spectrum of clonal hematopoiesis in VEXAS syndromeFernanda Gutierrez-Rodrigues, Yael Kusne, Jenna Fernandez, et al.Eclinicalmedicine|February 3, 2026
Efficacy, safety, and tolerability of arbaclofen in Autistic children and adolescents, the AIMS-2-TRIALS-CT1: a randomized, double-blind, placebo-controlled phase II trialM Parellada, A San José Cáceres, R Delorme, et al.Arthritis & Rheumatology (Hoboken, N.J.)|March 29, 2021
Somatic Mutations in UBA1 Define a Distinct Subset of Relapsing Polychondritis Patients With VEXASMarcela A Ferrada, Keith A Sikora, Yiming Luo, et al.Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|March 31, 2021
Phase I Study of Zotiraciclib in Combination with Temozolomide for Patients with Recurrent High-grade AstrocytomasJing Wu, Ying Yuan, Debra A Long Priel, et al.JAMA Oncology|July 17, 2015
Treatment With Carfilzomib-Lenalidomide-Dexamethasone With Lenalidomide Extension in Patients With Smoldering or Newly Diagnosed Multiple MyelomaNeha Korde, Mark Roschewski, Adriana Zingone, et al.Blood|April 20, 2021
SASH3 variants cause a novel form of X-linked combined immunodeficiency with immune dysregulationOttavia M Delmonte, Jenna R E Bergerson, Tomoki Kawai, et al.Blood|July 6, 2022
Translation of cytoplasmic UBA1 contributes to VEXAS syndrome pathogenesisMarcela A Ferrada, Sinisa Savic, Daniela Ospina Cardona, et al.Pageof 58