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‘…
NMDP Cycle is kicking off cycling events throughout the U.S. to raise funds to help eradicate barriers to treatment for patients, including those with sickle cell disease (SCD), who need a blood stem cell transplant. Formerly known as the Be The Match Tour de TC, NMDP Cycle…
Oxbryta (voxelotor), an oral medication approved to treat sickle cell disease (SCD), prevented children’s red blood cells from stiffening into a characteristic sickle-like shape and breaking down, possibly helping more oxygen to get into tissues in the body. That’s according to a study reporting data on 10 children…
The American Red Cross and the Sickle Cell Disease Association of Illinois (SCDAI) recently joined a national distributor of propane, natural gas, and other energy-related products to host a blood donation event to raise awareness about sickle cell disease and encourage blood donations to support transfusions for sickle cell…
Authorities in the U.K. have preliminarily recommended that Casgevy (exagamglogene autotemcel) not be reimbursed for eligible patients with sickle cell disease (SCD) under its national public health program based on uncertainties about the gene therapy’s cost-effectiveness. The drafted decision from the National Institute for Health and Care…
While children with sickle cell anemia — the most common form of sickle cell disease — are vulnerable to serious infections and stroke, many don’t receive the antibiotics or annual ultrasounds that national guidelines suggest could keep them healthier, a study found. Only about 1 in 5 children took…
Bluebird Bio has struck a deal with Medicaid in the state of Michigan to allow eligible people with sickle cell disease access to the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel). The new deal will provide Lyfgenia through an outcomes-based agreement — a financial structure in…
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…
People with sickle cell disease (SCD) or sickle cell trait (SCT) face an increased risk of hospitalization and mortality following infection with SARS-CoV-2, the virus that causes COVID-19, compared with the general population, according to a pooled analysis of published studies. Researchers also noted that kidney complications after COVID-19…
Suburban Propane joined the American Red Cross and other supporters in February to mark Black History Month with a blood drive that celebrated the life and work of Charles R. Drew, a renowned surgeon, medical researcher, and pioneer in blood transfusions. Drew was known as the “father…
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