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…
News
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 Improves Several SCD Features in Experimental Mice Models, Study Shows
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…
New Pre-transplant Treatment Regimen Improves Survival of Kids with Sickle Cell Disease, Trial Shows
A new pre-transplant conditioning regimen remarkably improves rejection rates and survival in children with sickle cell disease (SCD), according to the results of Phase 1/2 trial (NCT00920972). The study, “Unrelated Umbilical Cord Blood Transplantation for Sickle Cell Disease Following Reduced Intensity Conditioning: Results of a Phase I Trial,”…
Researchers have developed an accurate, low–cost potential test for diagnosing people with sickle cell anemia. The test, based on a lateral flow strip, is designed for use in areas where SCA treatment exists but testing methods are inaccessible, such as sub-Saharan Africa, where SCA is prominent. While researchers were optimistic about…
Multidisciplinary Care Team Greatly Reduces Risk in Pregnant SCD Patients in Low-income Countries
According to a study published in the American Journal of Hematology, the presence of a multidisciplinary clinical team can dramatically reduce maternal and perinatal mortality rates in pregnant women with sickle cell disease (SCD), especially in under-developed and low-income countries. In recent decades, medical advances have significantly improved the survival of those…
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