hereditary pyropoikilocytosis
MeSH: C563004ORPHA: 988671 Treatment Available
Overview
autosomal recessive inherited severe hemolytic anemia. It is a subtype of hereditary elliptocytosis and is characterized by partial spectrin deficiency
Available Treatments (1)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| eculizumab Orphan Cold Chain | IV infusion, 10mg/mL concentrate for solution | FDA Approved, EMA Approved | 9 | 14d |
Classification & Codes
MeSH Code
C563004Orphanet Code
ORPHA:98867hereditary pyropoikilocytosis
| MeSH | C563004 |
| Orphanet | ORPHA:98867 |
| Treatments | 1 drug(s) |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO