Multiple daily doses of GBT601, Global Blood Therapeutics (GBT)’s experimental oral therapy for sickle cell disease (SCD), were generally well tolerated and showed promising pharmacological and efficacy signals in six patients in a Phase 1 trial. These results support further evaluation of GBT601 as the potential successor to GBT’s…
News
The total lifetime medical costs of living with sickle cell disease (SCD) total about $1.6 million for women and $1.7 million for men younger than 65 who hold private health insurance in the U.S., a study found. Researchers concluded that some of these expenses — which are roughly four…
ARU-1801, Aruvant Sciences’ experimental gene therapy for sickle cell disease (SCD), led to the production of fetal hemoglobin in red blood cells and significantly lowered the number of vaso-occlusive crises (VOCs) in a group of five people with severe SCD, according to data from the ongoing Phase…
CRISPR Therapeutics and Vertex have launched two Phase 3 trials to assess the safety and effectiveness of CTX001, an experimental gene-editing cell therapy, one in children with sickle cell disease (SCD) and another for those with transfusion-dependent beta thalassemia (TDT). Both Phase 3 studies will enroll up to…
Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease (SCD) who are being treated with hydroxyurea. The company announced the software upgrade on May 8…
Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…
Robotic ducks were delivered recently as comfort companions to children with sickle cell disease (SCD) at the Children’s Regional Hospital at Cooper in South Jersey. The life-sized cuddly robot, called “My Special Aflac Duck,” were made available free to children, 3 and older, in a new collaboration between…
Exposure to systemic steroids — frequently used to treat asthma or ease inflammation — is significantly associated with a higher risk of painful vaso-occlusive crises (VOCs) leading to hospitalization in children and adults with sickle cell disease (SCD), a French study shows. “Individuals living with SCD often suffer…
While most adolescent boys and young men with sickle cell disease (SCD) report wanting biological children in the future, few are aware of the impact their disease and its treatments can have on their fertility, a small, pilot U.S. study suggests. Similar knowledge gaps were found for these patients’…
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