genetic cardiac disease

ORPHA: 980541 Treatment Available

Overview

rare genetic heart disease

Available Treatments (1)

DrugFormStatusCountriesLead Time
Ataluren
Orphan
Powder for oral suspensionEMA Approved521d

Classification & Codes

Orphanet Code

ORPHA:98054
genetic cardiac disease
OrphanetORPHA:98054
Treatments1 drug(s)
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO