Anemia
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Drug fails to meet endpoint in phase 3 ITP trial
The SYK inhibitor fostamatinib did not meet the primary endpoint in a phase 3 study of adults with chronic/persistent immune thrombocytopenia (ITP...
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Gene-editing approach is ‘important advance’ in SCD, doc says
sickle cell disease Image by Graham Beards Researchers have described a gene-editing technique that can correct the sickle cell mutation in...
From the Journals
Calcium channel blocker reduces cardiac iron loading in thalassemia major
Amlodipine is a promising addition to chelation in patients with cardiac siderosis.
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Inflammation may predict transformation to AML
Inflammatory signaling in mesenchymal niche cells can be used to predict the transformation from pre-leukemic syndrome to acute myeloid leukemia (...
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Lifestyle may impact life expectancy in mild SCD
alongside a normal one Image by Betty Pace A case series published in Blood indicates that some patients with mildly symptomatic sickle cell...
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Drug could reduce morbidity, mortality in aTTP, doc says
Photo courtesy of ASH THE HAGUE—Caplacizumab has the potential to reduce morbidity and mortality associated with acquired thrombotic...
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Study sheds light on platelet disorders
Image by Andre E.X.
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Gene therapy accepted into PRIME program
Photo by Chad McNeeley An investigational gene therapy known as LentiGlobin BB305 has been accepted into the European Medicines Agency’s (EMA’s)...
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Smartphone app measures hemoglobin
Photo by Daniel Sone HEIDELBERG, GERMANY—Scientists have developed a smartphone application called HemaApp that can be used to measure hemoglobin...
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FDA grants drug orphan designation for PNH
The US Food and Drug Administration (FDA) has granted orphan drug designation to Coversin as a treatment for paroxysmal nocturnal hemoglobinuria (...
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Gene therapy effective against SCD in mice
blood cells from a mouse Image courtesy of University of Michigan Preclinical research suggests a novel gene therapy may be effective against...