Lindsey Shapiro, PhD, science writer —

In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data. Developer Beam Therapeutics will present the findings at the upcoming American Society of Hematology…

Enlargement of the left atrium, one of the heart’s upper chambers, is associated with a greater sickle cell disease (SCD) burden for patients, with more emergency department visits and blood test abnormalities, according to a new study from the U.S. A larger left atrium is believed to be a…

People with sickle cell trait (SCT), those who carry a mutation in one copy of the HBB gene but do not have sickle cell disease (SCD), are at an increased risk of blood clots compared with people who don’t carry the trait regardless of racial or ethnic background, according…

A Massachusetts scientist has been awarded the seventh annual Trailblazer Prize for Clinician-Scientists by the Foundation for the National Institutes of Health (FNIH) for his research into understanding human blood cell development and how it’s altered in conditions such as sickle cell disease (SCD) — work that helped lead…

Seeking treatment for acute bouts of pain in hospitals and urgent care clinics is a significant source of distress for people with sickle cell disease (SCD), a report suggests. In interviews with SCD patients, scientists found patients’ distress went beyond the pain itself and included feelings of stigma and…

Longer gaps in transitioning from pediatric to adult care were associated with more hospital stays for people with sickle cell disease (SCD), a study indicates. Young adults who had a delay of more than six months were twice as likely to be hospitalized over those who made the switch…

Certain blood biomarkers of fat dysregulation, inflammation, and red blood cell destruction, known as hemolysis, may be used to predict the short-term risk of acute chest syndrome (ACS) in people with sickle cell disease (SCD). That’s according to a new study from France, in which scientists identified these…

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…

Authorities in the U.K. have preliminarily recommended that Casgevy (exagamglogene autotemcel) not be reimbursed for eligible patients with sickle cell disease (SCD) under its national public health program based on uncertainties about the gene therapy’s cost-effectiveness. The drafted decision from the National Institute for Health and Care…

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…