Women with sickle cell disease have high-risk pregnancies with significant potential for health crises and death; physicians are concerned that tighter restrictions on abortions may compromise their care.
A liquid biopsy test used for molecular profiling in patients with solid tumors also identified patients who were at risk for clonal hematopoiesis, which may develop into myeloid blood cancer.
Single-dose gene therapy, betibeglogene autotemcel, may represent a cure for most with beta-thalassemia, but it costs a record-high $2.8 million. Details on how coverage will work remain murky