Walker–Warburg syndrome

MeSH: D058494ORPHA: 8991 Treatment Available

Overview

rare form of autosomal recessive congenital muscular dystrophy

Available Treatments (1)

DrugFormStatusCountriesLead Time
idebenone
Orphan
oral tablet, 150 mgEMA Approved921d

Clinical Presentation

Signs and symptoms associated with Walker–Warburg syndrome, sourced from HPO and Orphanet clinical annotations.

AreflexiaPachygyriaCerebellar hypoplasiaMuscle weaknessSpecific learning disabilityLissencephalyAplasia/Hypoplasia involving the skeletal musculatureVentriculomegalyPolymicrogyriaAbnormality of neuronal migrationAbnormal cerebellar vermis morphologyAbnormal cortical gyrationSkeletal muscle atrophyMuscular dystrophyChorioretinal dysplasiaRetinal dysplasiaMetatarsus valgusAbnormal circulating aldolase concentrationAbnormal circulating creatine kinase concentrationAbnormal circulating lactate dehydrogenase concentrationCryptorchidismMacrocephalyGlaucomaAnophthalmiaMicrophthalmiaAgenesis of corpus callosumDandy-Walker malformationAbsent septum pellucidumCorneal opacityHypoplasia of penisCleft palateSubmucous cleft hard palateBifid uvulaMicrocephalyPosteriorly rotated earsLow-set earsProtruding earMicrocorneaCataractIris colobomaSeizureHydrocephalusRetinal detachmentRetinal dystrophyAbnormal optic nerve morphologyOptic atrophyIntellectual disabilityHypotoniaGlobal developmental delayHyporeflexia

Classification & Codes

MeSH Code

D058494

Orphanet Code

ORPHA:899
Walker–Warburg syndrome
MeSHD058494
OrphanetORPHA:899
Treatments1 drug(s)
Symptoms on record50 signs
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
Walker–Warburg syndrome | OrphanDrug