News

Treatment with delta-aminolevulinate (ALA), a precursor of heme — the part of hemoglobin that carries oxygen — could be a future therapy option for people with sickle cell anemia and β-thalassemia, a recent study suggests. The study, “δ-Aminolevulinate induces fetal hemoglobin expression by enhancing…

White blood cells, especially monocytes, may underlie the breathing difficulties affecting children with sickle cell disease (SCD), a small pilot study suggests. Asthma is estimated to impact from 17% to 28% of all children with SCD, its researchers noted, and obstructive lung disease — which affects exhaling — is…

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

Enhanced parenting skills, including attentive listening and engaging in conversations, may help improve cognitive functioning in children with sickle cell disease (SCD), a study suggests. Parent stress was related to lower parenting responsiveness, which also may lead to lower cognitive function in children, the researchers said. The study, “…

Caraphenol A — a small molecule closely related to a natural compound called resveratrol, which is produced by red grapes — increased the efficacy of hematopoietic stem cell-based gene therapy, a new study shows. The molecule holds potential to shorten the time and cost of using these gene therapies…

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…

Insufficient levels of selenium in patients with sickle cell disease (SCD) are associated with red blood cell destruction, or hemolysis, according to a study from Brazil. As such, the study authors recommend those patients should include selenium-rich foods in their diets. The study, “Selenium Status and…

The prenatal test UNITY, which uses maternal blood samples, is effective in screening for sickle cell disease (SCD), cystic fibrosis (CF), spinal muscular atrophy (SMA), and thalassemias, a study has found. The study, “A novel high-throughput molecular counting method with single base-pair resolution enables accurate…

The U.S. Food and Drug Administration (FDA) has awarded $2 million to support a Phase 2 clinical trial testing whether daily supplementation of vitamin D reduces the risk of respiratory complications in children and adolescents with sickle cell disease (SCD). Gary M. Brittenham, MD, a pediatrician…

A newly designed viral vector — the vehicle that delivers a gene therapy to a patient’s cells — for use in sickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models…