News

Being hospitalized due to a vaso-occlusive crisis (VOC) is associated with a significant reduction in quality of life for children with sickle cell disease (SCD), and has a special impact on their physical functioning, a study finds. The negative effects were particularly higher in the first months after…

An advisory committee to the U.S. Food and Drug Administration (FDA) has completed its meeting on exa-cel — formally exagamglogene autotemcel — ahead of the agency’s approval decision on the gene editing therapy, expected next month. The meeting was scheduled as part of the FDA’s review of exa-cel, which is…

If the U.S. Food and Drug Administration (FDA) approves the experimental sickle cell disease (SCD) gene therapy lovotibeglogene autotemcel (lovo-cel), its developer Bluebird Bio plans to sell the voucher it will receive that can be redeemed to speed up the review of a future application of a different…

Researchers at the University of Tennessee Health Science Center (UTHSC) have received a grant from the U.S. Department of Agriculture to support a specialized education program for improving the care of people with sickle cell disease (SCD) in medically underserved areas of the U.S. Led by principal investigator Sara…

A $12.2-million grant awarded to the New York-based Mount Sinai Health System will enable a nationwide observational study to help better personalize sickle cell disease (SCD) treatments. “Sickle cell traditionally has been a neglected disease, but it benefited from a flurry of innovation over the last decade and…

The number of red blood cells that produce the abnormal hemoglobin S (HbS), which characterizes sickle cell disease (SCD), is reduced by the experimental gene therapy BCH-BB694, as is their tendency to take a sickle-like shape. That’s according to single-cell data from seven patients in an ongoing Phase…

A portrait of 4-year-old Kynnedi Sturges, who is living with sickle cell disease (SCD) in Camden, Arkansas, was unveiled last week in an international art exhibit by the nonprofit Beyond the Diagnosis. Displaying artworks of 40 children with rare diseases, the exhibit is being hosted by…

The Georgia State University Research Foundation has received a grant from the Centers for Disease Control and Prevention (CDC) to support the ongoing Sickle Cell Data Collection (SCDC) program in that state. Established by the CDC in 2015, the program seeks to assess the frequency of…

To help heighten awareness of sickle cell disease (SCD) and pediatric cancers, while bringing comfort and relief to patients, the supplemental insurance provider Aflac recently gifted its My Special Aflac Duck social robot to children at the University of New Mexico (UNM) Children’s Hospital in Albuquerque. The delivery…

Rates of life-threatening infections by pneumococcal bacteria have significantly declined, by about 80%, among children with sickle cell disease (SCD) since the introduction of pneumococcal conjugate vaccines 20 years ago. That’s according to a study analyzing more than two decades of healthcare data from young SCD patients in metropolitan…