congenital muscular dystrophy with intellectual disability

ORPHA: 370968

Overview

human disease

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 congenital muscular dystrophy with intellectual disability, sourced from HPO and Orphanet clinical annotations.

Intellectual disabilityElevated circulating creatine kinase concentrationFloppy infantHypoglycosylation of alpha-dystroglycanReduced muscle fiber alpha dystroglycanMicrocephalyGlobal developmental delayMotor delayCerebral cortical atrophyMultiple joint contracturesLimb-girdle muscle weaknessPoor gross motor coordinationCalf muscle hypertrophyFeeding difficultiesFatigable weakness of skeletal musclesCryptorchidismMicropenisAbnormality of the eyeStrabismusMyopiaPigmentary retinopathyAbnormality of the nervous systemReduced tendon reflexesCerebellar vermis hypoplasiaCerebellar hypoplasiaHypoplasia of the corpus callosumRespiratory insufficiencyVentriculomegalyPoor speechLoss of ambulationAbnormal periventricular white matter morphologyScoliosisHip dislocationRespiratory failureAxial muscle weaknessAbnormality of connective tissueSkeletal muscle hypertrophyDecreased cervical spine mobilityAbnormal pons morphologyNeuropathic spinal arthropathyFacial palsySevere intellectual disabilityAbnormality of the tongue muscle

Classification & Codes

Orphanet Code

ORPHA:370968
congenital muscular dystrophy with intellectual disability
OrphanetORPHA:370968
Treatments0 drug(s)
Symptoms on record43 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO