ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that trigger the production of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and other blood disorders. SCD belongs to a group of inherited disorders known as hemoglobinopathies, primarily caused by mutations in…
News
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…
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…
A new way to monitor blood cells that doesn’t require microscopic imaging or biochemical markers may allow for better management of sickle cell disease, a study suggests. The study, “Electrical Impedance Characterization of Erythrocyte Response to Cyclic Hypoxia in Sickle Cell Disease,” was published in the…
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…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Wide interest in a CBS 60 Minutes story about a Florida woman with sickle cell disease (SCD) whose symptoms were eased through gene therapy has prompted the National Heart, Lung, and Blood Institute (NHLBI) to raise awareness of other work the National Institutes of Health…
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