Oxbryta (voxelotor), an oral medication approved to treat sickle cell disease (SCD), prevented children’s red blood cells from stiffening into a characteristic sickle-like shape and breaking down, possibly helping more oxygen to get into tissues in the body. That’s according to a study reporting data on 10 children…
While children with sickle cell anemia — the most common form of sickle cell disease — are vulnerable to serious infections and stroke, many don’t receive the antibiotics or annual ultrasounds that national guidelines suggest could keep them healthier, a study found. Only about 1 in 5 children took…
Nearly 1 in 3 children and teens with sickle cell disease (SCD) reports persistent or chronic pain, according to a new study that tested how well routine screening for pain worked as a tool to help identify those pediatric patients with the greatest need for interventions. “Pain screening fit…
A biomedical engineer at Kennesaw State University has been given a $426,000 grant to build a low-cost device helping to predict the risk of a stroke in children with sickle cell disease by using light to monitor blood flow to the brain. Paul Lee, PhD, an assistant professor at…
The first patient has been dosed in BEACON, a Phase 1/2 clinical trial of a transplant-based gene-editing stem cell therapy called BEAM-101 that Beam Therapeutics is developing for sickle cell disease (SCD). After the transplant, stem cells began to “engraft,” meaning they started to grow and give rise…
Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older. The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also…
A clinical project designed to test whether masitinib, AB Science’s experimental oral therapy for sickle cell disease (SCD), can resolve complications associated with the inherited disorder has been awarded €9.2 million (about $10 million) in public funding in France. The funding comes as part of a larger investment of €160…
Children and young adults with kidney failure due to sickle cell disease (SCD) wait nearly two times longer for a kidney transplant and have higher mortality than those whose kidney failure is not caused by SCD, a study found. “Children in the U.S. with SCD now have higher survival…
More than 1 in 3 children and adolescents with sickle cell disease (SCD) develop retinopathy, or damage to the blood vessels in the retina, the light-sensitive layer of tissue at the back of the eye, according to a new U.S. study. In addition, nearly one-tenth of eyes with retinopathy…
For nearly half of people with sickle cell disease who took part in the Phase 2 part of a clinical trial called RISE UP, treatment with the oral medication mitapivat brought about a significant increase in levels of hemoglobin — the oxygen-carrying protein in red blood cells — new…
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