adult-onset distal myopathy due to VCP mutation

ORPHA: 329478

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 adult-onset distal myopathy due to VCP mutation, sourced from HPO and Orphanet clinical annotations.

TremorFacial diplegiaBowel incontinenceBack painReduced vital capacityDistal muscle weaknessDementiaGait disturbanceAbnormality of the musculature of the lower limbsProgressive neurologic deteriorationFrequent fallsFasciculationsMyalgiaMuscle spasmEMG: myopathic abnormalitiesScapular wingingRimmed vacuolesMildly elevated creatine kinaseIntrinsic hand muscle atrophyNecrotizing myopathyWeakness of the intrinsic hand musclesFoot dorsiflexor weaknessFatty replacement of skeletal muscleUrinary incontinenceDepressionAnxietyDecreased nerve conduction velocityParkinsonism

Classification & Codes

Orphanet Code

ORPHA:329478
adult-onset distal myopathy due to VCP mutation
OrphanetORPHA:329478
Treatments0 drug(s)
Symptoms on record28 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO