A newly designed viral vector — the vehicle that delivers a gene therapy to a patient’s cells — for use in sickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models…
A community town hall is set for Detroit to raise awareness about sickle cell disease, with a discussion about current and future therapies, as part of the Sideline Sickle Cell campaign by Emmaus Life Sciences, together with the Sickle Cell Disease Association of America Michigan Chapter. The…
A medication used to treat high blood pressure, called valsartan, may help decrease the stickiness of red blood cells and reduce the risk of vaso-occlusive crisis in people with sickle cell disease (SCD), a research study suggests. The study, “Valsartan impedes epinephrine-induced ICAM-4 activation on normal, sickle cell…
An upcoming analysis by the Institute for Clinical and Economic Review (ICER) will compare the clinical efficacy and economic value of Novartis’ crizanlizumab and Global Blood Therapeutics‘ voxelotor, lead candidate therapies for sickle cell disease (SCD). ICER’s report will be discussed during its…
African-Americans with sickle cell trait, carriers for sickle cell but without the disease, have no greater risk of cognitive decline than other adults of similar age, an analysis of a large population study shows. The study “Sickle cell trait and risk of cognitive impairment in African-Americans: The REGARDS cohort” was…
Survival of sickle cell disease patients (SCD) with end-stage renal disease who undergo kidney transplant is significantly lower compared to other renal disease patients who have the same procedure, a retrospective analysis shows. That finding, “Survival and specific outcome of sickle cell disease patients after renal transplantation,” was…
The Phase 3 RESET trial testing the effectiveness and safety of rivipansel (GMI-1070), Pfizer‘s investigational candidate to treat vaso-occlusive crisis (VOC) in hospitalized patients with sickle cell disease (SCD), failed to reach both its primary and secondary goals, top-line study results show. “We are disappointed with the results, as we…
Children with sickle cell disease, like adults, have a distinct group of immune cells, called innate like T-cells, that appear to promote the chronic inflammation associated with SCD, researchers report. Their work “Innate-like T cells in children with sickle cell disease” was published in the journal PLOS…
A non-invasive prenatal test to diagnose a risk of sickle cell disease in a baby is possible and may be available in clinics in a near future, if further testing confirms its efficacy, researchers in the U.K. report. Julia van Campen presented the first results of the non-invasive test in…
Treatment with hydroxyurea since early infancy, starting at the age of one, is effective and safe at preventing sickle cell disease (SCD) complications, a follow-up study has found. The study “Prospective longitudinal follow-up of children with sickle cell disease treated with hydroxyurea since infancy” was published in journal…
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