News

The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 clinical trial. Findings suggest the treatment could help lower the risk of stroke in patients. The…

The first healthy volunteer has been dosed in a Phase 1 clinical study of CLY-124 — a novel oral small molecule that Cellarity is developing to treat sickle cell disease — following clearance of the company’s investigational new drug application by the U.S. Food and Drug Administration. “Initiation…

Higher pain scores at the time of discharge from the emergency department (ED) may predict the likelihood of hospitalization in children with sickle cell disease (SCD) who seek emergency care for acute pain episodes, according to a study in the U.S. The study also found that patients with severe…

Adults with sickle cell disease (SCD) who are started on buprenorphine to ease chronic pain — either in a clinical setting or at home via telemedicine — may be able to reduce their use of opioids and visit the hospital less frequently, a small study suggests. Buprenorphine induction —…

Most people with sickle cell disease (SCD) seem to meet or exceed current physical activity recommendations for the general population, a recent study in Senegal shows. Notably, greater step counts and time spent in more intense activities were associated with lower pain frequency and intensity, as well as lower…

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…