Hermansky-Pudlak syndrome with pulmonary fibrosis

ORPHA: 2315001 Treatment Available

Available Treatments (1)

DrugFormStatusCountriesLead Time
ruxolitinib
Orphan
Oral tablet, 5mg, 10mg, 15mg, 20mg, 25mg; Topical cream 1.5%FDA Approved117d

Classification & Codes

Orphanet Code

ORPHA:231500
Hermansky-Pudlak syndrome with pulmonary fibrosis
OrphanetORPHA:231500
Treatments1 drug(s)
Statuspublished

Treatment Summary

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