News

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…

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…

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…

The Oncologic Drug Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Emmaus Life Sciences’s New Drug Application (NDA) for Endari to treat sickle cell disease on May 24. “The Advisory Committee meeting is an important step forward in the review process for this promising therapy,”…

Researchers at New York’s Weill Cornell Medicine have discovered an innovative method of producing healthy stem cells that could significantly improve treatment of sickle cell anemia and other blood disorders. A study describing the method, “Conversion of adult endothelium to immunocompetent haematopoietic stem cells,” appeared in the journal Nature. Hematopoietic…

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to IMR-687, Imara’s drug candidate for the treatment of sickle cell disease (SCD). The investigational therapy has also been granted orphan drug status by the FDA. Rare pediatric disease designation is granted to drugs that show promise…

GlycoMimetics, in collaboration with Pfizer, is now enrolling participants for a Phase 3 clinical trial to test the effectiveness and safety of the drug candidate rivipansel (GMI-1070) for the treatment of vaso-occlusive crisis (VOC) in hospitalized patients with sickle cell disease (SCD). “We can report that the Phase…

Differences in comorbidities, pain, healthcare utilization and psychosocial outcomes were addressed in three groups of sickle cell disease (SCD) patients, distinguished by their respective ages, in the Pain in the Sickle Cell Epidemiology Study (PiSCES). Other studies are needed to determine if age-specific healthcare measures might improve these patients’ lives. The…