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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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…
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‘Sickle Cell Speaks’ Campaign Raises Awareness with Aim of Eroding Stigmas, GBT and Partners Say
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…
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…
The Sickle Cell Disease Association of Illinois (SCDAI) announced plans to hold its 45th Annual 8K Walk/Jog/Bike-A-Thon on July 20, continuing the group’s longstanding efforts to raise funds and disease awareness. Rain or shine, the event will take place starting at 8 a.m. along the shores of Lake…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
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