News

BEAM-101, a one-time, gene-edited cell therapy, continues to show durable therapeutic benefits more than one year after treatment in people with sickle cell disease (SCD), according to new data from the Phase 1/2 BEACON trial. The treatment’s developer, Beam Therapeutics, shared data from the first 17 patients dosed…

From illuminating public buildings in red to candlelight vigils and walks, supporters are gearing up to raise awareness and increase public knowledge about sickle cell disease (SCD) on June 19, World Sickle Cell Day. This year’s global theme is “Global Action, Local Impact: Empowering Communities for Effective Self-Advocacy.” SCD…

BEAM-101, a genetically modified cell therapy for sickle cell disease (SCD) that’s being tested in clinical trials, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA gives the designation to experimental therapies designed to treat conditions that affect fewer than 200,000 people…

The U.S. Food and Drug Administration has granted orphan drug status to Sanofi’s rilzabrutinib, an investigational therapy aimed at preventing painful vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). This designation intends to encourage companies to develop therapies for rare diseases, or those affecting fewer…

An algorithm that grew out of a collaboration between different specialties at the Medical University of South Carolina (MUSC) may help reduce unnecessary hospital admissions for children with sickle cell disease (SCD), according to a new study. “This algorithm safely allows our care of these patients to align with…

Adolescents and young adults with sickle cell disease (SCD) may face challenges in accessing physical therapy that could help them manage symptoms, a study reports. While young people with poorer quality-of-life metrics tended to receive more referrals to physical therapists, this did not translate to more physical therapy visits…

Meningitis — an inflammation of the protective membranes surrounding the brain and spinal cord — remains associated with a case-fatality rate of nearly 30% in children with sickle cell disease (SCD), according to the findings of a new two-decade study from France. This is despite vaccinations for SCD children…

Many adolescents with sickle cell disease (SCD) aren’t having recommended depression screenings nor regularly visiting their primary care providers (PCPs), even though most have a registered PCP, a study reports. “Despite a higher overall rate of depression in this population … most youth are not being screened for depression…

A program that provides integrated primary and specialist care for adults with sickle cell disease (SCD) significantly reduced emergency department visits, according to a study from Massachusetts General Hospital. “Our Comprehensive Sickle Cell Disease Center (CSCDC) is one of few comprehensive adult care models that integrates [primary care physicians]…

Sickle cell disease (SCD) and sickle cell trait (SCT) can increase the risk of eye diseases associated with diabetes mellitus, according to a recent study. Findings indicated patients who had diabetes along with SCT or SCD were more likely to have procedures to treat eye disease and poorer overall…