Charcot-Marie-Tooth disease axonal type 2K

ORPHA: 101097

Overview

Charcot-Marie-Tooth disease type 2 that has material basis in homozygous or compound heterozygous mutation in the GDAP1 gene on chromosome 8q

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 axonal type 2K, sourced from HPO and Orphanet clinical annotations.

Peripheral axonal degenerationImpaired vibratory sensationDistal sensory impairmentSkeletal muscle atrophyAxonal lossSensorimotor neuropathyDecreased number of small peripheral myelinated nerve fibersChronic axonal neuropathyImpaired pain sensationDistal upper limb muscle weaknessProximal lower limb muscle weaknessProximal upper limb muscle weaknessDistal lower limb muscle weaknessImpaired tactile sensationPeripheral demyelinationAbnormality of the vertebral columnSplit handAreflexiaReduced tendon reflexesFlexion contractureVocal cord paresisHoarse voicePes cavusTalipes equinovarusUnsteady gaitFrequent fallsLoss of ambulationQuadriceps muscle weaknessImpaired distal proprioceptionClusters of axonal regenerationIntrinsic hand muscle atrophyPeroneal muscle weaknessHand muscle weaknessImpaired tandem gaitAbnormal thorax morphologyMotor delay

Classification & Codes

Orphanet Code

ORPHA:101097
Charcot-Marie-Tooth disease axonal type 2K
OrphanetORPHA:101097
Treatments0 drug(s)
Symptoms on record36 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
Charcot-Marie-Tooth disease axonal type 2K | OrphanDrug