disorder of phospholipids, sphingolipids and fatty acids biosynthesis with central nervous system predominant involvement

ORPHA: 3523063 Treatments Available

Contents

Overview

human disease

Drug	Form	Status	Countries	Lead Time
eliglustat Orphan	Oral capsule, 84mg	FDA Approved, EMA Approved	7	10d
miglustat Orphan	Oral capsule, 100mg	FDA Approved, EMA Approved	9	14d
idebenone Orphan	oral tablet, 150 mg	EMA Approved	9	21d

Orphanet Code

ORPHA:352306

disorder of phospholipids, sphingolipids and fatty acids biosynthesis with central nervous system predominant involvement

Treatment Summary

Factual Authority

Last Updated3/20/2026

Clinical DataHPO · Orphanet

Drug DataFDA · EMA · CDSCO