Bluebird Bio has announced it’s completed collecting cells from the first sickle cell disease (SCD) patient receiving the gene therapy Lyfgenia (lovotibeglogene autotemcel), following its recent approval in the U.S. The cells were collected at Children’s National Hospital in Washington D.C., which is part of the company’s…
England’s National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending the country’s National Health Service (NHS) cover Pfizer’s Oxbryta (voxelotor) to treat sickle cell disease. “Today’s approval by NICE marks a significant milestone in sickle cell care, addressing decades of limited access to…
Explosive synchronization in the brain, wherein a small change in the brain network causes it to sync up and undergo a sudden shift in behavior, could be behind frequent pain crises in sickle cell disease, according to a study that may offer a way to better predict the disease’s…
A bone marrow transplant approach that doesn’t use high-dose chemotherapy to prevent an immune response against a donor’s stem cells showed high rates of event-free and overall survival among sickle cell disease (SCD) patients treated in a Phase 2 clinical trial. The approach, which also expands the eligibility of…
Having male adolescents with sickle cell disease (SCD) undergo fertility testing by semen analysis may not be feasible if it involves travel to a reproductive diagnostic center, according to a recent study. Despite having access to reproductive health education and compensation to cover transportation costs, only 15% of adolescents…
A nurse clinician-scientist and professor at the University of Toronto, in Canada, is being honored for her work in developing iCanCope, a mobile app that aims to help in sickle cell disease (SCD) pain management. Jennifer Stinson, PhD, was awarded the 2023 Barer-Flood Prize from the Canadian Institutes…
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‘…
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