Hermansky-Pudlak syndrome 2

MeSH: C537709ORPHA: 183678

Overview

Hermansky-Pudlak syndrome that has material basis in homozygous or compound heterozygous mutation in the gene encoding the beta-3A subunit of the AP3 complex (AP3B1) on chromosome 5q14.1

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Classification & Codes

MeSH Code

C537709

Orphanet Code

ORPHA:183678
Hermansky-Pudlak syndrome 2
MeSHC537709
OrphanetORPHA:183678
Treatments0 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
Hermansky-Pudlak syndrome 2 | OrphanDrug