News

Vertex, CRISPR complete BLA for US approval of exa-cel

Vertex Pharmaceuticals and CRISPR Therapeutics have completed an application to the U.S. Food and Drug Administration (FDA) seeking  approval of exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The rolling biologics license application, which was initiated late…

Extended-release formulation of Ferriprox now approved in Canada

Health Canada has approved an extended-release formulation of Ferriprox (deferiprone) to treat iron overload in people with sickle cell disease (SCD) and other blood disorders. The key benefit of the new formulation, according to the therapy’s maker, Chiesi Global Rare Diseases, is that it reduces the medication’s dosing…

Free new app aims to help SCD patients and families manage care

Salveo Therapeutics and the newsmagazine African Sickle Cell News and World Report — known as Sickle Cell News — are collaborating to promote a new free application (app) that seeks to empower sickle cell disease (SCD) patients to take more control of their health and wellbeing. The overarching aim…

SCDAA, 8 pharma companies team up to increase trial participation

The Sickle Cell Disease Association of America (SCDAA) is collaborating with several pharmaceutical companies in a new initiative aimed at increasing awareness of the blood disorder and promoting clinical trial participation. Along with the SCDAA, the Sickle Cell Disease Collaboration of Advocates for Research, Education, and Science — officially…