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Variable Clinical Features in a Large Family With Diamond Blackfan Anemia Caused by a Pathogenic Missense Mutation in RPS19
Discussion: This large family with DBA demonstrates the heterogeneity of phenotypes that can be seen within the same genotype. Most family members presented with steroid-responsive anemia in infancy and subtle congenital malformations, findings consistent with recent genotype-phenotype studies of RPS DBA. However, two family members were relatively unaffected, underscoring the importance of further studies to assess modifier genes, and epigenetic and/or environmental factors which may result in normal erythropoiesis despite underlying ribosome dysfunction. This large, multigenerational family highlights the need for indivi...
Source: Frontiers in Genetics - July 18, 2022 Category: Genetics & Stem Cells Source Type: research

Janssen to Present the Strength and Promise of its Hematologic Malignancies Portfolio and Pipeline at ASH 2021
RARITAN, N.J., November 4, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that more than 45 company-sponsored abstracts, including 11 oral presentations, plus more than 35 investigator-initiated studies will be featured at the American Society of Hematology (ASH) Annual Meeting and Exposition. ASH is taking place at the Georgia World Congress Center in Atlanta and virtually from December 11-14, 2021.“We are committed to advancing the science and treatment of hematologic malignancies and look forward to presenting the latest research from our robust portfolio and pipeline during ASH...
Source: Johnson and Johnson - November 5, 2021 Category: Pharmaceuticals Tags: Innovation Source Type: news

Efficacy and Safety of Pomalidomide in Combination with Prednisone in Patients with Myelofibrosis and Anemia -- Final Results of a Prospective Phase 2 Study
CONCLUSION:Pomalidomide with prednisone is safe therapy with good anti-anemia activity in patients with MF. It could lead to transfusion independence in one third of patients for a median duration of about 30 months. ClinicalTrials.gov Identifier: NCT00946270.Table 1.DisclosuresDaver: Alexion: Consultancy; ImmunoGen: Consultancy; Pfizer: Research Funding; Karyopharm: Research Funding; Otsuka: Consultancy; Novartis: Consultancy; ARIAD: Research Funding; Incyte: Consultancy; Pfizer: Consultancy; BMS: Research Funding; Sunesis: Research Funding; Daiichi-Sankyo: Research Funding; Sunesis: Consultancy; Kiromic: Research Funding...
Source: Blood - November 21, 2018 Category: Hematology Authors: Masarova, L., Daver, N. G., Kadia, T. M., Pemmaraju, N., Jabbour, E. J., Zhou, L., Pierce, S. A., Cortes, J. E., Kantarjian, H. M., Verstovsek, S. Tags: 634. Myeloproliferative Syndromes: Clinical: Poster I Source Type: research