X-linked mental retardation with cerebellar hypoplasia and distinctive facial appearance

MeSH: C537456ORPHA: 137831

Overview

X-linked intellectual deficit-cerebellar hypoplasia, also known as OPHN1 syndrome, is a rare syndromic form of cerebellar dysgenesis characterized by moderate to severe intellectual deficit and cerebellar abnormalities

Available Treatments (0)

No treatments linked yet

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

Clinical Presentation

Signs and symptoms associated with X-linked mental retardation with cerebellar hypoplasia and distinctive facial appearance, sourced from HPO and Orphanet clinical annotations.

Reduced social responsivenessIntellectual disabilityGlobal developmental delayCerebellar hypoplasiaStrabismusDeeply set eyeAutistic behaviorReduced eye contactAtaxiaHypotoniaGeneralized hypotoniaAbnormal facial shapeVentriculomegalyGeneralized myoclonic seizureAbnormal speech patternModerate intellectual disabilityFocal impaired awareness seizurePoor speechPartial absence of cerebellar vermisSevere intellectual disabilityTall chinMandibular prognathiaWide nasal bridgeProminent noseIntention tremorPostural instabilityEnlarged cisterna magnaCaudate atrophyAplasia/Hypoplasia of the cerebellar vermisFrontal cortical atrophyRetrocerebellar cystAttention deficit hyperactivity disorderTemporal cortical atrophy

Classification & Codes

MeSH Code

C537456

Orphanet Code

ORPHA:137831
X-linked mental retardation with cerebellar hypoplasia and distinctive facial appearance
MeSHC537456
OrphanetORPHA:137831
Treatments0 drug(s)
Symptoms on record33 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO