509. Bone Marrow Failure and Cancer Predisposition Syndromes: Congenital: Poster I

Exploring the spectrum of non malignant complications in germline DDX41 associated hematologic disorders

Unsupervised clustering of DDX41 mutants informs on the somatic landscape of single germline hits

Decoding DDX41 Clinical impact of germline and somatic mutations in 77 high risk myeloid neoplasm patients

First in class small molecule calmodulin pathway modulators attenuate excess p53 activity and correct erythropoietic defects in models of diamond blackfan anemia DBA

ANKRD26 related thrombocytopenia Hematologic malignancy characteristics clinical outcomes and precursor states

Granulocyte maturation and splicing defects in DDX41 mutated leukemia

A zebrafish model of parn deficiency identifies altered lipid metabolism as a novel therapeutic target for restoring hematopoiesis in dyskeratosis congenita

A pilot study of low dose sirolimus to increase hematopoietic function in patients with RUNX1 familial platelet disorder