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Neuronopathy, distal hereditary motor, autosomal dominant 1(HMN1; DHMN1)

MedGen UID:
356618
Concept ID:
C1866784
Disease or Syndrome
Synonyms: CHARCOT-MARIE-TOOTH DISEASE, SPINAL, I; HMN I; NEURONOPATHY, DISTAL HEREDITARY MOTOR, HARDING TYPE I; Neuronopathy, distal hereditary motor, type 1; Neuronopathy, distal hereditary motor, type I; NEUROPATHY, DISTAL HEREDITARY MOTOR, HARDING TYPE I; SPINAL MUSCULAR ATROPHY, DISTAL, JUVENILE, AUTOSOMAL DOMINANT, HARDING TYPE I
SNOMED CT: Distal hereditary motor neuropathy type 1 (770630005); Autosomal dominant distal juvenile spinal muscular atrophy type 1 (770630005)
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.
 
Monarch Initiative: MONDO:0008451
OMIM®: 182960
Orphanet: ORPHA139518

Definition

Distal hereditary motor neuronopathy (dHMN or HMN) is a heterogeneous group of neuromuscular disorders caused by anterior horn cell degeneration and characterized by progressive distal motor weakness and muscular atrophy of the peripheral nervous system without sensory impairment. Distal HMN is also referred to as spinal Charcot-Marie-Tooth disease (spinal CMT). Distal HMN is often referred to as a 'neuronopathy' instead of a 'neuropathy' based on the hypothesis that the primary pathologic process resides in the neuron cell body and not in the axons (Irobi et al., 2006). Historically, Harding (1993) proposed a clinical classification of distal HMN into 7 phenotypic subtypes according to age at onset, mode of inheritance, and presence of additional features; see NOMENCLATURE. Genetic Heterogeneity of Autosomal Dominant Distal Hereditary Motor Neuronopathy Genetically distinct forms of autosomal dominant distal hereditary motor neuropathy include HMND1; HMND2 (158590), caused by mutation in the HSPB8 gene (608014); HMND3 (608634), caused by mutation in the HSPB1 gene (602195); HMND4 (613376), caused by mutation in the HSPB3 gene (604624); HMND5 (600794), caused by mutation in the GARS gene (600287); HMND6 (615575), caused by mutation in the FBXO38 gene (608533); HMND7 (158580), caused by mutation in the SLC5A7 gene (608761); HMND8 (600175), caused by mutation in the TRPV4 gene (605427); HMND9 (617721), caused by mutation in the WARS gene (191050); HMND10 (620080), caused by mutation in the EMILIN1 gene (130660); HMND11 (620528), caused by mutation in the SPTAN1 gene (182810); HMND12 (614751), caused by mutation in the REEP1 gene (609139); HMND13 (619112), caused by mutation in the BSCL2 gene (606158); and HMND14 (607641), caused by mutation in the DCTN1 gene (601143). See also X-linked HMN (HMNX; 300489), caused by mutation in the ATP7A gene (300011) on chromosome Xq21. Additional disorders with overlapping features include autosomal dominant ALS4 (602433), caused by mutation in the SETX gene (608465); and CMS7A (616040), caused by mutation in the SYT2 gene (600104). [from OMIM]

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).
Hammertoe
MedGen UID:
209712
Concept ID:
C1136179
Anatomical Abnormality
Hyperextension of the metatarsal-phalangeal joint with hyperflexion of the proximal interphalangeal (PIP) joint.
Upper limb muscle weakness
MedGen UID:
305607
Concept ID:
C1698196
Finding
Weakness of the muscles of the arms.
Babinski sign
MedGen UID:
19708
Concept ID:
C0034935
Finding
Upturning of the big toe (and sometimes fanning of the other toes) in response to stimulation of the sole of the foot. If the Babinski sign is present it can indicate damage to the corticospinal tract.
Impaired vibration sensation at ankles
MedGen UID:
343107
Concept ID:
C1854372
Finding
A decrease in the ability to perceive vibration at the ankles. Clinically, this is usually tested with a tuning fork which vibrates at 128 Hz and is applied to the malleoli of the ankles.
Chronic axonal neuropathy
MedGen UID:
867220
Concept ID:
C4021578
Disease or Syndrome
An abnormality characterized by chronic impairment of the normal functioning of the axons.
Hypertonia
MedGen UID:
10132
Concept ID:
C0026826
Finding
A condition in which there is increased muscle tone so that arms or legs, for example, are stiff and difficult to move.
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.

Recent clinical studies

Etiology

Cutrupi AN, Narayanan RK, Perez-Siles G, Grosz BR, Lai K, Boyling A, Ellis M, Lin RCY, Neumann B, Mao D, Uesugi M, Nicholson GA, Vucic S, Saporta MA, Kennerson ML
Brain 2023 Mar 1;146(3):880-897. doi: 10.1093/brain/awac424. PMID: 36380488Free PMC Article

Diagnosis

Lee AJ, Nam DE, Choi YJ, Nam SH, Choi BO, Chung KW
Genes Genomics 2020 Jun;42(6):663-672. Epub 2020 Apr 20 doi: 10.1007/s13258-020-00933-9. PMID: 32314272

Prognosis

Lee AJ, Nam DE, Choi YJ, Nam SH, Choi BO, Chung KW
Genes Genomics 2020 Jun;42(6):663-672. Epub 2020 Apr 20 doi: 10.1007/s13258-020-00933-9. PMID: 32314272

Clinical prediction guides

Lee AJ, Nam DE, Choi YJ, Nam SH, Choi BO, Chung KW
Genes Genomics 2020 Jun;42(6):663-672. Epub 2020 Apr 20 doi: 10.1007/s13258-020-00933-9. PMID: 32314272
Kamada M, Kawarai T, Miyamoto R, Kawakita R, Tojima Y, Montecchiani C, D'Onofrio L, Caltagirone C, Orlacchio A, Kaji R
Parkinsonism Relat Disord 2018 Jan;46:79-83. Epub 2017 Oct 21 doi: 10.1016/j.parkreldis.2017.10.012. PMID: 29107646
Brusse E, Majoor-Krakauer D, de Graaf BM, Visser GH, Swagemakers S, Boon AJ, Oostra BA, Bertoli-Avella AM
Neurogenetics 2009 Oct;10(4):289-97. Epub 2009 Apr 24 doi: 10.1007/s10048-009-0193-1. PMID: 19396477Free PMC Article
McEntagart M, Norton N, Williams H, Teare MD, Dunstan M, Baker P, Houlden H, Reilly M, Wood N, Harper PS, Futreal PA, Williams N, Rahman N
Am J Hum Genet 2001 May;68(5):1270-6. Epub 2001 Apr 4 doi: 10.1086/320122. PMID: 11294660Free PMC Article

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