syndromic neurometabolic disease with non-X-linked intellectual disability

ORPHA: 1820733 Treatments Available

Contents

Overview

human disease

Drug	Form	Status	Countries	Lead Time
Vigabatrin Orphan	Oral tablet 500mg; Oral powder for solution 500mg sachets	FDA Approved, EMA Approved	6	14d
Carnitine Orphan	Oral tablet 330mg; Oral solution 100mg/mL; IV injection 200mg/mL	FDA Approved, EMA Approved	9	7d
Levetiracetam	Oral tablet 250mg, 500mg, 750mg, 1000mg; Oral solution 100mg/mL; IV infusion 500mg/5mL	FDA Approved, EMA Approved	11	3d

Orphanet Code

ORPHA:182073

syndromic neurometabolic disease with non-X-linked intellectual disability

Treatment Summary

Factual Authority

Last Updated3/20/2026

Clinical DataHPO · Orphanet

Drug DataFDA · EMA · CDSCO