Casgevy (exagamglogene autotemcel) will be reimbursed by Germany’s public health system, enabling people with severe sickle cell disease (SCD) in the country, ages 12 and older, to access the one-time gene-editing therapy at low or no cost. The reimbursement agreement, established with Vertex Pharmaceuticals — which developed…
News
Sickle cell disease (SCD) hospitalization patterns vary across New York State, with New York City showing the largest share of hospitalizations and Long Island reporting the highest charges for care, a recent study showed. In addition, longer hospital stays were seen in regions where access to specialized care may…
A discovery about red blood cells could have implications for sickle cell disease (SCD) and other blood disorders marked by problems with hemoglobin, the protein red blood cells use to carry oxygen through the bloodstream. Scientists found that red blood cells growing under stressful conditions can import heme, an…
Women with sickle cell anemia (SCA), the most common and severe form of sickle cell disease (SCD), have significantly lower ovarian reserve relative to women without SCA and with proven fertility, a study in Nigeria shows. Ovarian reserve is a marker-based estimate of the remaining supply of eggs in the…
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…
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…
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