News Related to Primary Immunodeficiencies

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Rare Disease Therapies Advance for Dravet Syndrome and Spinal Muscular Atrophy

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.

Leonard Zon Wins 2026 March of Dimes Prize for Developmental Biology Research

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.