Leukotriene C4 synthase deficiency

MeSH: C565439ORPHA: 79507

Overview

Leukotriene C4 synthase deficiency is an extremely rare fatal neurometabolic developmental disorder characterized clinically by muscular hypotonia, psychomotor retardation, failure to thrive, and microcephaly

Available Treatments (0)

No treatments linked yet

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

Classification & Codes

MeSH Code

C565439

Orphanet Code

ORPHA:79507
Leukotriene C4 synthase deficiency
MeSHC565439
OrphanetORPHA:79507
Treatments0 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
Leukotriene C4 synthase deficiency | OrphanDrug