hemoglobin E-beta-thalassemia syndrome
ORPHA: 2312492 Treatments Available
Available Treatments (2)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| luspatercept Orphan Cold Chain | subcutaneous injection, 25 mg/vial, 75 mg/vial powder for solution | FDA Approved, EMA Approved | 8 | 10d |
| deferasirox Orphan | oral tablet, 125 mg, 250 mg, 500 mg; oral granules for suspension | FDA Approved, EMA Approved | 10 | 5d |
Clinical Presentation
Signs and symptoms associated with hemoglobin E-beta-thalassemia syndrome, sourced from HPO and Orphanet clinical annotations.
AnemiaImmunodeficiencyIncreased circulating ferritin concentrationAbnormal hemoglobin
Classification & Codes
Orphanet Code
ORPHA:231249hemoglobin E-beta-thalassemia syndrome
| Orphanet | ORPHA:231249 |
| Treatments | 2 drug(s) |
| Symptoms on record | 4 signs |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO