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Charcot-Marie-Tooth disease type 1F(CMT1F)

MedGen UID:
334337
Concept ID:
C1843164
Disease or Syndrome
Synonyms: Charcot-Marie-Tooth disease, demyelinating, type 1f; Charcot-Marie-Tooth Neuropathy Type 1F/2E; CHARCOT-MARIE-TOOTH NEUROPATHY, TYPE 1F; CMT 1F
SNOMED CT: Charcot-Marie-Tooth disease type IF (719980006); Charcot-Marie-Tooth disease type 1F (719980006)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Gene (location): NEFL (8p21.2)
 
Monarch Initiative: MONDO:0011902
OMIM®: 607734
Orphanet: ORPHA101085

Definition

A form of Charcot-Marie-Tooth disease type 1, with a variable clinical presentation that can range from severe impairment with onset in childhood to mild impairment appearing during adulthood. The disease has characteristics of progressive peripheral motor and sensory neuropathy with distal paresis in the lower limbs that varies from mild weakness to complete paralysis of the distal muscle groups, absent tendon reflexes and reduced nerve conduction. Caused by mutations in the NEFL gene (8p21.2). [from SNOMEDCT_US]

Clinical features

From HPO
Pes cavus
MedGen UID:
675590
Concept ID:
C0728829
Congenital Abnormality
An increase in height of the medial longitudinal arch of the foot that does not flatten on weight bearing (i.e., a distinctly hollow form of the sole of the foot when it is bearing weight).
Dysphagia
MedGen UID:
41440
Concept ID:
C0011168
Disease or Syndrome
Difficulty in swallowing.
Tremor
MedGen UID:
21635
Concept ID:
C0040822
Sign or Symptom
An unintentional, oscillating to-and-fro muscle movement about a joint axis.
Areflexia
MedGen UID:
115943
Concept ID:
C0234146
Finding
Absence of neurologic reflexes such as the knee-jerk reaction.
Global developmental delay
MedGen UID:
107838
Concept ID:
C0557874
Finding
A delay in the achievement of motor or mental milestones in the domains of development of a child, including motor skills, speech and language, cognitive skills, and social and emotional skills. This term should only be used to describe children younger than five years of age.
Hyporeflexia
MedGen UID:
195967
Concept ID:
C0700078
Finding
Reduction of neurologic reflexes such as the knee-jerk reaction.
Segmental peripheral demyelination/remyelination
MedGen UID:
335873
Concept ID:
C1843077
Finding
A segmental pattern of demyelination and regeneration (remyelination) affecting peripheral nerves.
Myelin outfoldings
MedGen UID:
334341
Concept ID:
C1843168
Finding
The presence of excessive redundant myelin in the peripheral nerve sheath.
Clusters of axonal regeneration
MedGen UID:
335897
Concept ID:
C1843169
Finding
Groups of small caliber axons in peripheral nerve biospies indicative of axonal regeneration.
Distal sensory impairment
MedGen UID:
335722
Concept ID:
C1847584
Finding
An abnormal reduction in sensation in the distal portions of the extremities.
Onion bulb formation
MedGen UID:
376237
Concept ID:
C1847906
Finding
Repeated episodes of segmental demyelination and remyelination lead to the accumulation of supernumerary Schwann cells around axons, which is referred to as onion bulb formation. This finding affects peripheral nerves.
Motor delay
MedGen UID:
381392
Concept ID:
C1854301
Finding
A type of Developmental delay characterized by a delay in acquiring motor skills.
Decreased number of peripheral myelinated nerve fibers
MedGen UID:
346872
Concept ID:
C1858285
Finding
A loss of myelinated nerve fibers in the peripheral nervous system (in general, this finding can be observed on nerve biopsy).
Decreased motor nerve conduction velocity
MedGen UID:
388130
Concept ID:
C1858729
Finding
A type of decreased nerve conduction velocity that affects the motor neuron.
Distal muscle weakness
MedGen UID:
140883
Concept ID:
C0427065
Finding
Reduced strength of the musculature of the distal extremities.
Distal amyotrophy
MedGen UID:
338530
Concept ID:
C1848736
Disease or Syndrome
Muscular atrophy affecting muscles in the distal portions of the extremities.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
Follow this link to review classifications for Charcot-Marie-Tooth disease type 1F in Orphanet.

Recent clinical studies

Diagnosis

Kim HJ, Kim SB, Kim HS, Kwon HM, Park JH, Lee AJ, Lim SO, Nam SH, Hong YB, Chung KW, Choi BO
Mol Genet Genomic Med 2022 Feb;10(2):e1870. Epub 2022 Jan 19 doi: 10.1002/mgg3.1870. PMID: 35044100Free PMC Article

Clinical prediction guides

Stone EJ, Uchida A, Brown A
Cytoskeleton (Hoboken) 2019 Jul;76(7-8):423-439. Epub 2019 Nov 6 doi: 10.1002/cm.21566. PMID: 31574566Free PMC Article

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