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.
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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07251179 |
Characterization of Autoreactive b Lymphocytes in Autoimmune Diseases and Immune Deficiencies |
NOT_YET_RECRUITING | |
| NCT02349906 |
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases |
COMPLETED | PHASE2 |
| NCT01953016 |
Participation in a Research Registry for Immune Disorders |
COMPLETED |
