Mulago launched a 20-month project to screen Ugandan children for Inborn Errors of Immunity. Studies found 901 variants in uncharacterized genes and 815 variants in humoral IEI genes in Africa.
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 |
|---|---|---|---|
| NCT07766304 |
Long-term Psychological and Cognitive Evaluation of Children Treated With Allogeneic Hematopoietic Stem Cell Transplantation for Immunodeficiency |
NOT_YET_RECRUITING | |
| NCT07251179 |
Characterization of Autoreactive b Lymphocytes in Autoimmune Diseases and Immune Deficiencies |
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 |
