The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Forma Therapeutics‘ FT-4202 for the treatment of sickle cell disease (SCD) This designation is given to investigational therapies intended to treat rare conditions (those that affect fewer than 200,000 people in the U.S.). It provides companies…
Children and adolescents with sickle cell disease (SCD) who lack vitamin D are hospitalized more often due to pain crises, have longer hospital stays, and make more frequent visits to the emergency room, a new study has found. The study, “The association between vitamin D…
The American Society of Hematology released new clinical guidelines on the use of blood transfusions in people with sickle cell disease (SCD). The transfusion guidelines are the second in a series of five therapy guidelines ASH is developing for SCD. Guidelines for managing heart and kidney disease…
Voxelotor can reduce the severity of existing leg ulcers and the incidence of new ones in people with sickle cell disease (SCD), new clinical trial results suggest. The findings detailed by the treatment’s developer, Global Blood Therapeutics (GBT), are being presented at the 13th Annual Academy for Sickle Cell and Thalassemia…
Children with sickle cell disease (SCD) who arrive at an emergency department with vaso-occlusive episodes (VOEs) are more likely to have a longer delay before treatment, receive less narcotics than recommended, and to be admitted to the hospital, compared with SCD patients who present to a specialty day hospital, a…
A new way to monitor blood cells that doesn’t require microscopic imaging or biochemical markers may allow for better management of sickle cell disease, a study suggests. The study, “Electrical Impedance Characterization of Erythrocyte Response to Cyclic Hypoxia in Sickle Cell Disease,” was published in the…
Global Blood Therapeutics (GBT) announced the five organizations that will receive more than $200,000 worth of grants through its new Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL). ACCEL was launched in February by the clinical-stage biopharmaceutical company with the goal of improving the…
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