Novartis is recalling its sickle cell disease (SCD) treatment Adakveo (crizanlizumab) in the U.K., after the nation’s drug regulatory agency revoked the conditional approval it had previously granted to the antibody-based therapy. The U.K. Medicines & Healthcare products Regulatory Agency (MHRA) made its decision based on Adakveo’s failure…
A national £1.5 million ($1.9 million) technology investment in the U.K. is aiming to expand access to red blood cell exchange devices for people with sickle cell disease (SCD) living in North East England. The focus will be on improving treatment availability for underserved patients in urban, rural, and…
Nearly 1 in 3 children and teens with sickle cell disease (SCD) reports persistent or chronic pain, according to a new study that tested how well routine screening for pain worked as a tool to help identify those pediatric patients with the greatest need for interventions. “Pain screening fit…
Nearly 3 in every 4 people with sickle cell disease (SCD) in the U.S. are not using an approved, disease-modifying therapy (DMT), according to a nationwide claims analysis. These patients tend to be older and to have relatively milder disease — the lowest prevalence of vaso-occlusive crises (VOCs)…
The European Commission has granted conditional approval to the gene-editing therapy Casgevy (exagamglogene autotemcel) for patients ages 12 and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), two blood disorders. Eligible patients include those with SCD who experience recurrent vaso-occlusive crises (VOCs) and…
Heavier menstrual bleeding and pain during menstrual periods appear to be prevalent among adolescents and young women with sickle cell disease (SCD), affecting their quality of life, but few of these patients use hormonal therapies to help regulate their cycles, a single-site U.S. survey study reported. Knowledge of what…
Treatment with hydroxyurea reduced the incidence of severe or invasive infections by 60% among children in Uganda with sickle cell anemia, the most common and usually the most severe form of sickle cell disease, a recent study reports. “Our investigation provides powerful…
Starting in 2025, the U.S. Department of Health and Human Services (HHS) will introduce a pricing model that aims to make new cell and gene therapies more accessible for people with sickle cell disease who are on Medicaid, the health program for lower income residents jointly funded by the…
England’s National Health Service (NHS) is introducing a new genetic test that seeks to make it safer for people with sickle cell disease and other blood disorders to receive transfusions. “Thousands of people living with sickle cell disease and thalassaemia will be eligible for this new world-first blood…
Comer Children’s Hospital at the University of Chicago will be one of the first centers in the U.S. to offer new gene therapies for sickle cell disease that were approved last year by the U.S. Food and Drug Administration (FDA). “I’m excited on behalf…
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