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Mar 05, 2026
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.
Feb 17, 2026
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.
Feb 14, 2026
Disc Medicine received a complete response letter from the FDA for bitopertin as a treatment for erythropoietic protoporphyria. The FDA acknowledged the drug lowers PPIX levels but requires evidence from the ongoing Phase 3 APOLLO study before approval.
