Charcot-Marie-Tooth disease X-linked dominant 1

ORPHA: 101075

Overview

Charcot-Marie-Tooth disease X-linked that has material basis in hemizygous or heterozygous mutation in the GJB1 gene on chromosome Xq13

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 Charcot-Marie-Tooth disease X-linked dominant 1, sourced from HPO and Orphanet clinical annotations.

Sensory neuropathyAreflexiaPes cavusDistal upper limb amyotrophyDistal lower limb amyotrophyAbnormal nerve conduction velocityImpaired pain sensationHearing impairmentAtaxiaDysarthriaExcessive daytime somnolenceGait disturbanceTremorLanguage impairmentScoliosisKyphosis

Classification & Codes

Orphanet Code

ORPHA:101075
Charcot-Marie-Tooth disease X-linked dominant 1
OrphanetORPHA:101075
Treatments0 drug(s)
Symptoms on record16 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO