Marisa Wexler, MS, senior science writer —

Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle cell disease (SCD), with better outcomes when blood markers are matched between donor and recipient, a study suggests. The study, “The role of HLA matching in unrelated donor hematopoietic stem cell…

FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected biological effects, providing preliminary support of efficacy, according to data from an ongoing Phase 1 clinical trial. Forma Therapeutics, which is developing FT-4202, plans to start a global Phase…

Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for treating hemolytic anemia in people with sickle cell disease (SCD) who are 12 or older. The submission is anticipated by…

Global Blood Therapeutics (GBT) will ask the U.S. Food and Drug Administration (FDA) to allow children with sickle cell disease (SCD), ages 4 to 11, to also be treated with Oxbryta (voxelotor). The company announced plans to file an application requesting extended approval after a Type B meeting with FDA…

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…