Ribosome biogenesis and protein synthesis are fundamental rate-limiting steps for cell
growth and proliferation. The ribosomal proteins (RPs), comprising the structural parts of the …

Cancer is driven by multiple types of genetic alterations, which range in size from point
mutations to whole-chromosome gains and losses, known as aneuploidy. Chromosome …

Background Risk stratification and therapeutic decision-making for myelodysplastic
syndromes (MDS) are based on the International Prognostic Scoring System–Revised (IPSS …

Somatic mutations in UBA1 cause vacuoles, E1 ubiquitin-activating enzyme, X-linked,
autoinflammatory somatic (VEXAS) syndrome, an adult-onset inflammatory disease with an …

Glasdegib is a Hedgehog pathway inhibitor. This phase II, randomized, open-label,
multicenter study (ClinicalTrials. gov, NCT01546038) evaluated the efficacy of glasdegib …

Background Patients with anemia and lower-risk myelodysplastic syndromes in whom
erythropoiesis-stimulating agent therapy is not effective generally become dependent on red …

Disease overview The myelodysplastic syndromes (MDS) are a very heterogeneous group
of myeloid disorders characterized by peripheral blood cytopenias and increased risk of …

We conducted a phase I clinical trial of H3B-8800, an oral small molecule that binds Splicing
Factor 3B1 (SF3B1), in patients with MDS, CMML, or AML. Among 84 enrolled patients (42 …

This study investigated the efficacy and safety of azacitidine maintenance in the
posttransplant setting based on the encouraging phase 1/2 reports for azacitidine …

Purpose The optimal regimen intensity before allogeneic hematopoietic cell transplantation
(HCT) is unknown. We hypothesized that lower treatment-related mortality (TRM) with …