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Human Mutation|April 14, 2025
Genotype/Phenotype Relationship: Lessons From 137 Patients With PMM2-CDGSander Pajusalu, Mari-Anne Vals, Mercedes Serrano, et al.
Journal of Inherited Metabolic Disease|August 21, 2021
Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatmentEugen Mengel, Marc C Patterson, Rosalia M Da Riol, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 3, 2021
Leukemic extracellular vesicles induce chimeric antigen receptor T cell dysfunction in chronic lymphocytic leukemiaMichelle J Cox, Fabrice Lucien, Reona Sakemura, et al.
Cancer Research Communications|May 4, 2026
MRI Deep Learning for Differentiating Glioblastoma, IDH-Wildtype from Central Nervous System Diffuse Large B-cell LymphomaMana Moassefi, Paul A Decker, Gian Marco Conte, et al.
Blood|January 28, 2022
Targeting cancer-associated fibroblasts in the bone marrow prevents resistance to CART-cell therapy in multiple myelomaReona Sakemura, Mehrdad Hefazi, Elizabeth L Siegler, et al.
Molecular Therapy. Oncology|June 3, 2025
Intrinsic immunosuppressive features of monocytes suppress CAR-T19 through IL-1 pathway modulation in mantle cell lymphomaKun Yun, R Leo Sakemura, Ismail Can, et al.
Cancer Immunology Research|July 10, 2021
Development of a Clinically Relevant Reporter for Chimeric Antigen Receptor T-cell Expansion, Trafficking, and ToxicityReona Sakemura, Aditya Bansal, Elizabeth L Siegler, et al.
The New England Journal of Medicine|July 12, 2023
BRAF-MEK Inhibition in Newly Diagnosed Papillary CraniopharyngiomasPriscilla K Brastianos, Erin Twohy, Susan Geyer, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|December 23, 2024
Longitudinal Glioma Monitoring via Cerebrospinal Fluid Cell-Free DNACecile Riviere-Cazaux, Xiaoxi Dong, Wei Mo, et al.
Blood|October 25, 2023
CD19 occupancy with tafasitamab increases therapeutic index of CART19 cell therapy and diminishes severity of CRSR Leo Sakemura, Claudia Manriquez Roman, Paulina Horvei, et al.
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