News

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…

Strong evidence points to some complications of sickle cell anemia (SCA) having as much to do with nutrition as they do with genes.  Characteristics and symptoms of the disease include poor growth, pain, infection, decreased muscle function, problems with cognition and coordination, low antioxidant status, and anemia.

The Centers for Disease Control and Prevention (CDC) will share an award total of nearly $1.2 million with institutions in seven U.S. states, allowing them to take part in a data gathering program on sickle cell disease and how it affects daily life. The funding brings to nine…

A multidisciplinary plan implemented at Yale New Haven Children’s Hospital was able to cut by more than half the number of days young sickle cell disease (SCD) patients were hospitalized for a vaso-occlusive crisis and lower their readmission rates, a study reports.

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…