syndrome with combined immunodeficiency
ORPHA: 3312173 Treatments Available
Overview
human disease
Available Treatments (3)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| Berotralstat Orphan | Capsule | FDA Approved | 4 | 14d |
| ruxolitinib Orphan | Oral tablet, 5mg, 10mg, 15mg, 20mg, 25mg; Topical cream 1.5% | FDA Approved | 11 | 7d |
| C1 esterase inhibitor (human) Orphan Cold Chain | Lyophilized powder for reconstitution | FDA Approved | 4 | 7d |
Classification & Codes
Orphanet Code
ORPHA:331217syndrome with combined immunodeficiency
| Orphanet | ORPHA:331217 |
| Treatments | 3 drug(s) |
| Status | published |
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO