Patricia Inácio, PhD, science writer —

Mitem Pharma has acquired worldwide rights to Desferal (deferoxamine), a therapy commonly used to lower excessive and damaging iron levels induced by blood transfusions in people with sickle cell disease (SCD) or other anemias. The acquisition was reached in an agreement between Mitem and Novartis, which…

The NewYork-Presbyterian Dalio Center for Health Justice is using basketball to raise awareness about sickle cell disease (SCD). The Sickle Cell Awareness Ball campaign features a special-edition basketball with one crescent-shaped red panel, representing the cell that’s a hallmark feature of the disease. The black-and-red ball is featured…

Supplementing with vitamin D for six months safely improved bone health and functional capacity in children and adolescents with sickle cell disease (SCD), a study finds. More studies are required to assess the “optimal dose, duration of supplementation, long-term [adverse events], and efficacy in different types of SCD, wrote…

The 2024 Shaw Prize in Life Science & Medicine has been awarded to two U.S. scientists whose discoveries paved the way for the development of gene therapies for sickle cell disease (SCD) and other blood disorders. This year’s prize — carrying a gold medal and a $1.2 million award…

Health Canada has granted priority review to the application of the gene-editing therapy exagamglogene autotemcel (exa-cel) for patients ages 12 and older with sickle cell disease (SCD) having recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The agency is expected to complete the review of Vertex Pharmaceuticals‘…

Tim Randolph, PhD, a scientist and professor at Saint Louis University in St. Louis, has been named a senior member of the U.S. National Academy of Inventors (NAI) for his work to develop low-cost diagnostic tools for sickle cell disease. The NAI Senior Members Program recognizes the spirit…

A $3-million federal grant to investigate the potential of repurposing a blood pressure medication for sickle cell disease (SCD)-related cardiac disease has been awarded to researchers at Indiana University School of Medicine. The grant from the U.S. Department of Defense was given to the study’s lead researcher Ankit A.

A month after its U.S. approval, Lyfgenia (lovotibeglogene autotemcel), Bluebird Bio’s gene therapy for sickle cell disease (SCD), is readying to treat patients there. According to the company, 35 out of 48 qualified treatment centers across the U.S. started accepting patient referrals as of Jan. 5. Treatment…

Being hospitalized due to a vaso-occlusive crisis (VOC) is associated with a significant reduction in quality of life for children with sickle cell disease (SCD), and has a special impact on their physical functioning, a study finds. The negative effects were particularly higher in the first months after…

Editas Medicine has expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, its experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. If approved, the scaling up of EDIT-301 needs to follow current good manufacturing practice (cGMP) standards — set…