News

The American Institutes for Research (AIR) has been awarded a $28 million federal contract to help make cell and gene therapy (CGT) treatments more accessible to Medicaid recipients, initially focusing on sickle cell disease (SCD). The contract, from the Center for Medicare and Medicaid Innovation in the Centers for…

Reproductive health issues were found to be common after a stem cell transplant among young people with sickle cell disease (SCD), most of whom underwent the procedure at approximately 11 years of age, according to a recent analysis of STELLAR data. The observed reproductive health issues, which included abnormalities in…

Children with sickle cell disease (SCD) may be more likely than their peers to have attention-deficit hyperactivity disorder (ADHD), according to a new study that found that the prevalence of ADHD in SCD kids may be more than double the expected rate in the general population. Based on these…

Enrollment has been completed in the adolescent group of the Phase 1/2/3 RUBY clinical trial testing Editas Medicine’s gene-editing therapy, renizgamglogene autogedtemcel (reni-cel), in people with sickle cell disease (SCD). “I am proud of the Editas team’s work and our advancement in the first half of 2024 as…

A bone marrow transplant may help reverse heart damage in people with sickle cell disease (SCD), according to a study. The findings were published in a letter to the editor titled, “Impact of Hematopoietic Cell Transplantation on Myocardial Fibrosis in Young Patients with Sickle Cell Disease,” in…

A novel small molecule called SR-18292, which acts to boost the production of fetal hemoglobin, was shown to reduce disease severity in a mouse model of sickle cell disease (SCD) and demonstrated benefits in other lab testing in a new study. “We’ve uncovered a promising approach that can offer…

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…

The U.S. Food and Drug Administration (FDA) gave the greenlight for Iomab-ACT, a potentially less toxic conditioning regimen, to be tested in people with sickle cell disease (SCD) undergoing a stem cell transplant. The study will be conducted in collaboration between Actinium Pharmaceuticals, which is developing Iomab-ACT,…

Hydroxyurea does not reduce ovarian reserve, or the quantity and quality of eggs in the ovaries, in girls and young women with sickle cell disease (SCD), according to a direct analysis of ovarian tissue. These findings suggest measures to preserve fertility may be unnecessary before hydroxyurea treatment. “This…

The use of eculizumab, an immunosuppressive medication approved for several autoimmune diseases, was found to be safe and effective for treating hyperhemolysis syndrome or HHS — a serious complication of blood transfusions — in two pediatric patients with sickle cell disease (SCD), a study showed. HHS, which can be…