Anemia
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EMA recommends orphan status for drug in SCD
and normal red blood cells Image by Graham Beards The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has issued...
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Gene-editing method cures thalassemia in mice
Photo by Aaron Logan A new gene-editing strategy may be able to cure thalassemia, according to preclinical research published in Nature...
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Test approved to screen donated blood for sickle cell trait
The US Food and Drug Administration (FDA) has approved use of the PreciseType HEA test to screen blood donors for sickle cell trait (SCT). The...
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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)...