Riociguat, a medication used to treat forms of pulmonary hypertension, was found to safely lower blood pressure in people with sickle cell disease (SCD) who had risk factors for poor outcomes, according to findings from an early clinical trial. “Our results are encouraging, and open the door to…
A nurse training program at the University of Tennessee Health Science Center (UTHSC) is expanding to train more nurses to care for people with sickle cell disease (SCD). Called the Sickle Cell Boot Camp to Promote Nursing Excellence Nationally and Globally Utilizing a Train the Trainer Model,…
The National Institutes of Health (NIH) has awarded a $6.1 million grant to the Feinstein Institutes for Medical Research to advance knowledge of red cell disorders, such as sickle cell disease (SCD), to develop new and better treatments. SCD is caused by genetic mutations that ultimately result…
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…
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