News

Randomized controlled trials are needed to examine the benefits of vaccinating people with sickle cell disease (SCD) against salmonella infections, which can be particularly fatal to children with the disease, a study says. The review study, “Vaccines for preventing invasive salmonella infections in …

Crizanlizumab (SEG101), Novartis‘ investigational compound for sickle cell disease (SCD), received breakthrough therapy designation from the U.S. Food and Drug Administration for its potential to prevent vaso-occlusive crises (VOCs). The FDA designation was granted based on promising data from the company’s multicenter, randomized, double-blind, 52-week, Phase…

The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease. A Phase 1/2 clinical trial (NCT03745287) is recruiting patients. CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells. Fetal hemoglobin is…

The U.S. opioid epidemic is not linked to higher in-hospital mortality in patients with sickle cell disease (SCD), according to a national study. The study came about due to the pronounced increase in opioid-related overdoses and deaths in the U.S. that has been reported since 2000. Its researchers did…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Bluebird bio presented new positive results from the ongoing Phase 1/2 HGB-206 clinical trial evaluating the effectiveness and safety of investigational gene therapy LentiGlobin in patients with severe sickle cell anemia. The company had previously revealed findings from the trial at the end of last year and earlier this…

Global Blood Therapeutics’ lead therapeutic candidate voxelotor can induce rapid, robust, and sustained improvements in the amount of hemoglobin in adolescents and adults with sickle cell disease (SCD), according to interim results from the first part of a Phase 3 trial. The latest results of GBT’s clinical…

Hemolytic transfusion reactions, a serious complication of red blood cell transfusion, can be difficult to diagnose in patients with sickle cell disease and even be misinterpreted as severe vaso-occlusive crisis. A report published in the journal Transfusion highlights the importance of accurate and prompt diagnosis of such complications in…

For this year’s Solutions to Empower Patients (STEP) program, global healthcare company Novartis has announced it will fund up to five original and creative ideas to help sickle cell disease (SCD) patients and their families. Each selected proposal will be awarded up to $50,000. Specifically, Novartis is seeking…

A new clinical decision support-based tool can help primary care physicians improve the care and management of patients with sickle cell disease (SCD), a study reports. The study, “Effectiveness of Clinical Decision Support Based Intervention in the Improvement of Care for Adult Sickle Cell Disease Patients in…