hemolytic anemia due to diphosphoglycerate mutase deficiency
ORPHA: 7141 Treatment Available
Overview
rare, autosomal recessive, inherited disorder caused by mutation of the BPGM gene. It is characterized by hemolytic anemia and splenomegaly
Available Treatments (1)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| folic acid | oral tablet 0.4mg, 0.8mg, 1mg, 5mg; injectable solution 5mg/mL | FDA Approved | 11 | 2d |
Classification & Codes
Orphanet Code
ORPHA:714hemolytic anemia due to diphosphoglycerate mutase deficiency
| Orphanet | ORPHA:714 |
| Treatments | 1 drug(s) |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO