News

Global Blood Therapeutics has expanded its ongoing HOPE-KIDS 1 trial (NCT02850406), evaluating the investigative GBT440 in pediatric patients with sickle cell disease, to include a new single-dose cohort in younger children ages 6 to 11. GBT440 is being developed as an oral, once-daily drug candidate for the treatment…

Endari (L-glutamine) today became the first therapy in almost 20 years to win approval from the U.S. Food and Drug Administration to treat sickle cell disease. In giving the green light to Endari use in patients starting at age 5, the FDA also made the oral drug the first ever…

Patients with sickle cell anemia who do not have alpha-thalassemia and the genetic rs1427407 T variant of the BCL11A gene may be at higher risk for hemolysis (rupture of red blood cells) and stroke, according to the results of studies of three independent groups. These findings were featured in…

The European Medicines Agency (EMA) has granted GBT440 — Global Blood Therapeutics’ lead drug candidate for sickle cell disease (SCD) — access to its Priority Medicines (PRIME) program. GBT440 is an oral, once-daily drug being developed to specifically inhibit hemoglobin polymerization and the consequent sickling of red blood cells, the hallmark…

Researchers at Mississippi State University (MSU) are using tiny polymer chips to build models to help them better understand sickle cell disease. Research on the models, which can also be used to study heart disease, might lead  identification of molecules or factors that new treatments can target to lessen organ damage…

Moderate exercise training can improve many features of sickle cell disease (SCD) in experimental mice models, including inflammation, lack of oxygen in the blood and spleen enlargement, according to a French study. The report, “Moderate exercise training decreases inflammation in transgenic sickle cell mice,” appeared in the scientific journal Blood…

The Health Resources and Services Administration (HRSA), part of the U.S. Department of Health and Human Services, has awarded the Sickle Cell Disease Association of America (SCDAA) a $2.9 million annual grant for the next four years to establish a national infrastructure to ensure sickle cell disease (SCD) patients receive appropriate follow–up care…

Several psychological factors such as depression, anxiety, and catastrophizing (believing something is worse than it is) can influence the perception and experience of pain in pediatric patients with sickle cell disease, according to a study published in The Journal of Pain.

A study reported that both PROMIS and ASCQ-Me measures are useful tools to monitor the daily functioning and well-being of adults living with sickle cell disease (SCD), but most ASCQ-Me scores were better predictors of SCD disease severity. The study, published in the journal Health and Quality of Life…

A study of children with sickle cell disease (SCD) referred for an evaluation of neuropsychological deficits found that while 19 of 89 patients (25%) were diagnosed with attention deficit hyperactivity disorder (ADHD), only 21% with ADHD had been prescribed medication for it. The study, “Attention Deficit Hyperactivity Disorder in…