One of the $3 million 2026 Breakthrough Prizes — in Life Sciences — has been awarded to two researchers whose discoveries reshaped the treatment of sickle cell disease (SCD). The award from the Breakthrough Prize Foundation, which counts its mission as “honoring scientists as heroes of our society,” is…
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A global, late-stage clinical trial testing etavopivat in people with sickle cell disease (SCD) has hit its main goals, showing that the experimental oral therapy improved hemoglobin levels and reduced the risk of painful episodes compared with a placebo. Based on these positive results from the Phase 3…
People with sickle cell disease (SCD) who live in sub-Saharan Africa are less likely than those in other countries to use common medications to manage the condition, including over-the-counter pain-relieving medicines, a study found. These patients also tended to report a lower perceived disease burden than those in high-income…
Clinician bias toward people with sickle cell disease (SCD) may be tied to stigma around opioid use, rather than race or chronic pain alone, a new study suggests. “Although patients with sickle cell disease routinely use opioid medications to manage their chronic pain, the vast majority do not…
Children with sickle cell anemia (SCA), the most common and severe form of sickle cell disease (SCD), show signs of altered development in specific regions of the brain, even in children without a history of silent or overt stroke, a new study from the U.S. reports. Using an artificial…
Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end. The application is expected to be supported by now-published interim data from…
Agios Pharmaceuticals is planning to file an application with the U.S. Food and Drug Administration (FDA) in the coming months that will seek accelerated approval of its oral therapy mitapivat for sickle cell disease (SCD). This decision by Agios follows a prefiling meeting with the regulatory agency in…
The Japan Agency for Medical Research and Development (AMED) — a national funding agency created to support medical R&D in the Asian nation — has awarded up to $32 million to Epifrontier Therapeutics to advance the clinical development of EPF-001, its investigational therapy for sickle cell disease (SCD)…
Through a project aimed at building empathy and understanding among clinicians about sickle cell disease (SCD)-related pain episodes, a team of nurses in Chicago has helped standardize emergency care for these complications and reduce door-to-painkiller time, according to a study. “I set out on this initiative with a very…
A bipartisan team of U.S. senators has introduced legislation to renew funding for a national initiative that connects people with blood disorders such as sickle cell disease (SCD) with unrelated donors for stem cell transplants. If the legislation passes, the national initiative, known as the C.W. Bill Young Cell…
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