mitochondrial disorder due to a defect in assembly or maturation of the respiratory chain complexes
ORPHA: 3091362 Treatments Available
Overview
human disease
Available Treatments (2)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| Sodium Phenylbutyrate Orphan | Tablets, Powder | FDA Approved | 4 | — |
| triheptanoin Orphan | oral liquid, 1000mg/mL | FDA Approved, EMA Approved | 6 | 21d |
Classification & Codes
Orphanet Code
ORPHA:309136mitochondrial disorder due to a defect in assembly or maturation of the respiratory chain complexes
| Orphanet | ORPHA:309136 |
| Treatments | 2 drug(s) |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO