New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
Researchers develop multiple strategies to enhance CAR T-cell therapy, including dual PGE2 receptor knockout for solid tumors and small-molecule switches using venetoclax and caffeine to control cellular activity and improve safety.
March of Dimes awarded the 2026 Richard B Johnston Jr, MD Prize to hematologist Leonard Zon for pioneering zebrafish research that illuminated blood development in embryos and led to breakthrough treatments for genetic blood disorders in children.
Researchers from Stanford Medicine developed an injection that reprograms cells to regrow cartilage by blocking the protein 15-PGDH, offering potential treatment for osteoarthritis from aging or injury.
Researchers have developed modified CAR T cells that overcome prostaglandin E2 suppression in solid tumors and a selective therapy targeting IGHV4-34 that spares healthy immune cells, addressing key limitations in current treatments.
