News

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…

Emmaus Life Sciences, accompanied by the Emmy Award-winning National Football League (NFL) broadcaster Solomon Wilcots, took its Sideline Sickle Cell campaign to the recent 6th Annual Sickle Cell Disease Patient & Family Educational Symposium that took place in Plano, Texas. Hosted the Sickle Cell Association of Texas…

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…

To lessen the need for hospitalizations and slow or reverse the organ damage seen in sickle cell disease (SCD), scientists at University of Pittsburgh (Pitt) will use a $19.2 million National Institutes of Health grant to test red blood cell exchange transfusion in patients. Co-led by the…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

The U.S. Food and Drug Administration (FDA) accepted Novartis’ Biologics License Application (BLA) seeking marketing permission for crizanlizumab (SEG101), a potential treatment for vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The agency granted the file priority review, to accelerate the product’s evaluation. “The…

In partnership with community organizations, Global Blood Therapeutics (GBT) is directing a national campaign to raising awareness and understanding of sickle cell disease, including among health professionals focused largely on pain crises, with the goal improving patient care. Called Sickle Cell Speaks, the campaign highlights diverse stories told…