Sickle cell anemia is a form of sickle cell disease, an inherited hemoglobin disorder in which red blood cells become hard, sticky, and sickle-shaped. These abnormal cells can obstruct blood flow and cause pain episodes and multi-organ complications.
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 |
|---|---|---|---|
| NCT07356050 |
Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease |
RECRUITING | |
| NCT07282210 |
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype |
NOT_YET_RECRUITING | PHASE2 |
| NCT07277023 |
The Influence of micro-and Macro Vascular Dysfunction on Clinical Severity in Adults With Sickle Cell Anemia (SS) and Sickle Cell Hemoglobin C Disease (SC) |
COMPLETED | NA |
| NCT07116772 |
Serial Assessment of Fertility Experiences |
RECRUITING | |
| NCT07094646 |
Full Assessment of Clinical Transfusion Support |
COMPLETED | |
| NCT06831799 |
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 |
COMPLETED | |
| NCT06698120 |
Awake Prone Positioning for Severe Acute Chest Syndrome |
NOT_YET_RECRUITING | NA |
| NCT06529874 |
Pressure Pain Tolerance in Relation to Balance and Strength in Children |
RECRUITING | NA |
| NCT06503458 |
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants |
TERMINATED | |
| NCT06016634 |
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease |
RECRUITING | PHASE2 |
