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Síndrome Roussy-Lévy
ORPHA:3115CID-10 · G60.0CID-11 · 8C20.YOMIM 180800DOENÇA RARA

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Introdução

O que você precisa saber de cara

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Síndrome Roussy-Lévy é uma neuropatia hereditária autossômica dominante, associada a genes como PMP22 e MPZ. Manifesta-se com nistagmo, arreflexia, ataxia da marcha, quedas frequentes e tremor postural, resultando em instabilidade e incoordenação motora.

Publicações científicas
52 artigos
Último publicado: 2024 Nov 27

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
Childhood
+ infancy
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: G60.0
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (2)
0202010694
Sequenciamento completo do exoma (WES)genetic_test
0301070040
Atendimento em reabilitação — doenças rarasrehabilitation
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🧠
Neurológico
8 sintomas
💪
Músculos
4 sintomas
🦴
Ossos e articulações
3 sintomas
👁️
Olhos
1 sintomas
😀
Face
1 sintomas
🫘
Rins
1 sintomas

+ 23 sintomas em outras categorias

Características mais comuns

55%prev.
Nistagmo
Frequente (79-30%)
55%prev.
Arreflexia
Frequente (79-30%)
55%prev.
Sensação vibratória prejudicada
Frequente (79-30%)
55%prev.
Ataxia da marcha
Frequente (79-30%)
55%prev.
Tremor postural
Frequente (79-30%)
55%prev.
Distúrbio da marcha
Frequente (79-30%)
42sintomas
Frequente (21)
Ocasional (6)
Sem dados (15)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 42 características clínicas mais associadas, ordenadas por frequência.

NistagmoNystagmus
Frequente (79-30%)55%
ArreflexiaAreflexia
Frequente (79-30%)55%
Sensação vibratória prejudicadaImpaired vibratory sensation
Frequente (79-30%)55%
Ataxia da marchaGait ataxia
Frequente (79-30%)55%
Tremor posturalPostural tremor
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa2desde 2024
Total histórico52PubMed
Últimos 10 anos7publicações
Pico20242 papers
Linha do tempo
2024Hoje · 2026🧪 1997Primeiro ensaio clínico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

2 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant.

PMP22Peroxisomal membrane protein 2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Seems to be involved in pore-forming activity and may contribute to the unspecific permeability of the peroxisomal membrane

LOCALIZAÇÃO

Peroxisome membrane

VIAS BIOLÓGICAS (1)
EGR2 and SOX10-mediated initiation of Schwann cell myelination
EXPRESSÃO TECIDUAL(Ubíquo)
Nervo tibial
3229.9 TPM
Tecido adiposo
421.5 TPM
Cervix Ectocervix
415.8 TPM
Fallopian Tube
379.8 TPM
Cervix Endocervix
354.6 TPM
OUTRAS DOENÇAS (7)
Roussy-Levy syndromeCharcot-Marie-Tooth disease type 3hereditary neuropathy with liability to pressure palsiesCharcot-Marie-Tooth disease type 1E
HGNC:9118UniProt:Q9NR77
MPZMyelin protein P0Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Is an adhesion molecule necessary for normal myelination in the peripheral nervous system. It mediates adhesion between adjacent myelin wraps and ultimately drives myelin compaction

LOCALIZAÇÃO

Cell membraneMyelin membrane

VIAS BIOLÓGICAS (1)
EGR2 and SOX10-mediated initiation of Schwann cell myelination
MECANISMO DE DOENÇA

Charcot-Marie-Tooth disease, demyelinating, type 1B

A dominant demyelinating form of Charcot-Marie-Tooth disease, a disorder of the peripheral nervous system, characterized by progressive weakness and atrophy, initially of the peroneal muscles and later of the distal muscles of the arms. Charcot-Marie-Tooth disease is classified in two main groups on the basis of electrophysiologic properties and histopathology: primary peripheral demyelinating neuropathies (designated CMT1 when they are dominantly inherited) and primary peripheral axonal neuropathies (CMT2). Demyelinating neuropathies are characterized by severely reduced nerve conduction velocities (less than 38 m/sec), segmental demyelination and remyelination with onion bulb formations on nerve biopsy, slowly progressive distal muscle atrophy and weakness, absent deep tendon reflexes, and hollow feet.

EXPRESSÃO TECIDUAL(Ubíquo)
Nervo tibial
5300.7 TPM
Cólon sigmoide
34.0 TPM
Esôfago - Junção
31.7 TPM
Esôfago - Muscular
30.5 TPM
Artéria coronária
19.6 TPM
OUTRAS DOENÇAS (9)
Roussy-Levy syndromeneuropathy, congenital hypomyelinating, 2Charcot-Marie-Tooth disease dominant intermediate DCharcot-Marie-Tooth disease type 2I
HGNC:7225UniProt:P25189

Variantes genéticas (ClinVar)

642 variantes patogênicas registradas no ClinVar.

🧬 MPZ: NM_000530.8(MPZ):c.118G>A (p.Gly40Ser) ()
🧬 MPZ: NM_000530.8(MPZ):c.111G>C (p.Glu37Asp) ()
🧬 MPZ: NM_000530.8(MPZ):c.101_106del (p.Thr34_Asp35del) ()
🧬 MPZ: NM_000530.8(MPZ):c.59_63dup (p.Val23fs) ()
🧬 MPZ: NM_000530.8(MPZ):c.408_412dup (p.Lys138fs) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 61 variantes classificadas pelo ClinVar.

18
43
Patogênica (29.5%)
VUS (70.5%)
VARIANTES MAIS SIGNIFICATIVAS
PMP22: NM_000304.4(PMP22):c.201del (p.Thr68fs) [Likely pathogenic]
MPZ: NM_000530.8(MPZ):c.30del (p.Ser11fs) [Pathogenic]
MPZ: NM_000530.8(MPZ):c.392dup (p.Asn131fs) [Likely pathogenic]
MPZ: NM_000530.8(MPZ):c.*360C>G [Conflicting classifications of pathogenicity]
MPZ: NM_000530.8(MPZ):c.173T>A (p.Val58Asp) [Conflicting classifications of pathogenicity]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Síndrome Roussy-Lévy

🗺️

Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

Nenhum ensaio clínico registrado para esta condição.

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Publicações mais relevantes

Timeline de publicações
7 papers (10 anos)
#1

Genetically confirmed Charcot-Marie-Tooth disease type 2A manifesting with postural tremor: a case report.

Journal of medical case reports2024 Nov 27

Charcot-Marie-Tooth disease is a spectrum of inherited disorders characterized by both motor and sensory manifestations, which include prominent distal muscle weakness, foot deformities (pes cavus and hammer toes), and sensory deficits. Postural tremor as a manifestation of Charcot-Marie-Tooth is seldom present, except in a variant of Charcot-Marie-Tooth subtype 1 (Roussy-Levy syndrome), and its presence often results in a diagnostic dilemma. We present a 34-year-old Eritrean man who came to our hospital with a complaint of tremors of the hands of 6 months duration. Associated with this, he had difficulty walking and weakness of the distal extremities bilaterally, prominently involving the lower limbs. The patient denied a family history of such illness. Physical examination revealed distal muscle weakness (4+/5 on upper limbs, while 3/5 on lower limbs bilaterally), pes cavus deformity, absent ankle reflexes, and mild vibratory sensory loss. We noted a postural tremor that attenuated when the patient assumed an anatomic position. The tremor was limited to the hands. Nerve conduction study of upper and lower limbs showed moderate to severe motor axonal and demyelinating polyneuropathy (axonal > demyelinating), suggestive of mixed axonal and demyelinating hereditary polyneuropathy. Subsequently, genetic testing revealed copy number changes (heterozygous deletion) on the MPZ and MFN2, while the PMP22 gene showed ambiguous copy number changes (decrease) on exons 2 and 3. Tying the clinical, electrophysiologic, and genetic findings, consideration of Charcot-Marie-Tooth subtype 2A with postural tremor was made. Subsequently, the patient was managed with regular physiotherapy and an anxiolytic resulting in minimal symptom improvement. The present case describes a 34-year-old male patient with Charcot-Marie-Tooth subtype 2A presenting with neuropathic postural tremor, which is a rare presentation of a common hereditary polyneuropathy. This case highlights the fact that tremors can be associated with peripheral neuropathy syndromes, and a high index of suspicion is needed to rightly diagnose our patients.

#2

Roussy-Lévy Syndrome: Pes Cavus, Tendon Areflexia, Amyotrophy, Gait Ataxia, and Upper Limb Tremor in a Patient with CMT Neuropathy.

Tremor and other hyperkinetic movements (New York, N.Y.)2024

Roussy-Lévy syndrome (RLS) is characterized by postural hand tremor seen in patients with familial autosomal dominant Charcot-Marie-Tooth (CMT) neuropathy. This video demonstrates irregular, jerky bilateral kinetic, postural, rest tremor affecting the right > left hand, along with pes cavus and gait ataxia in a patient with CMT disease. Pes cavus, tendon areflexia, sensory ataxia, and upper limb tremor should prompt consideration of CMT neuropathy. This video abstract depicts a bilateral hand tremor characteristic of Roussy-Lévy syndrome seen in patients with Charcot-Marie-Tooth disease neuropathy. The significance of the abstract lies in the phenomenology and the physiology of the tremor seen in patients with genetically confirmed duplication of PMP22 gene.

#3

Characterisation of Patients with SH3TC2 Associated Neuropathy in an Indian Cohort.

Neurology India2023

SH3TC2 variations lead to demyelinating recessive Charcot-Marie-Tooth (CMT) disease, which is commonly associated with early-onset scoliosis and cranial neuropathy. Data from Indian ethnicity is limited. We aim to report the characteristics of patients with SH3TC2-associated neuropathy from an Indian cohort. Data of five unrelated subjects with SH3TC2 variations were analyzed. Clinical features included female predominance (n = 4), early-onset neuropathy (n = 2), pes cavus and hammer toes (n = 4), kyphoscoliosis (n = 1), impaired vision and hearing (n = 1), facial muscle weakness (n = 1), impaired kinaesthetic sense (n = 3), tremor (n = 2), and ataxia (n = 1). Four patients had the "CMT" phenotype, while one patient had Roussy-Levy syndrome. All had demyelinating electrophysiology with conduction velocities being "very slow" in one, "slow" in one, "mildly slow" in two, and "intermediate" in one patient. Brain stem auditory evoked potentials were universally abnormal though only one patient had symptomatic deafness. Seven variants were identified in SH3TC2 [homozygous = 3 (c.1412del, c.69del, c.3152G>A), heterozygous = 4 (c.1105C>T, c.3511C>T, c.2028G>C, c.254A>T)]. Except for c.3511C>T variant, the rest were novel. Three patients had additional variations in genes having pathobiological relevance in other CMTs or amyotrophic lateral sclerosis. We provide data on a cohort of patients of Indian origin with SH3TC2 variations and highlight differences from other cohorts. Though the majority were not symptomatic for hearing impairment, evoked potentials disclosed abnormalities in all. Further studies are required to establish the functional consequences of novel variants and their interacting molecular partners identified in the present study to strengthen their association with the phenotype.

#4

Roussy-Lévy syndrome: a case of genotype-phenotype correlation.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2021 Oct
#5

Mutational screening of the SH3TC2 gene in Greek patients with suspected demyelinating recessive Charcot-Marie-Tooth disease reveals a varied and unusual phenotypic spectrum.

Journal of the peripheral nervous system : JPNS2019 Mar

Charcot-Marie-Tooth disease type 4 C (CMT4C) is an autosomal recessive form of demyelinating peripheral neuropathy caused by mutations in SH3TC2, characterized by early onset, spine deformities, and cranial nerve involvement. We screened SH3TC2 in 50 unrelated Greek patients with suspected demyelinating Charcot-Marie-Tooth disease and pedigree compatible with recessive inheritance. All patients had been previously screened for PMP22, GJB1, and MPZ mutations. We found five previously identified pathogenic mutations in SH3TC2 distributed among 13 patients in homozygosity or compound heterozygosity (p. Arg954Stop, Arg1109Stop, Gln892Stop, Ala878Asp, and Arg648Trp). Although most cases had early onset and spine deformities were almost omnipresent, a wide phenotypic spectrum was observed. Particularly notable were two siblings with Roussy-Lévy syndrome and one patient with young-onset trigeminal neuralgia. In conclusion, mutations in SH3TC2 are responsible for 26% of Greek patients with suspected CMT4, identifying CMT4C as the most common recessive demyelinating neuropathy in the Greek population, in accordance with other Mediterranean cohorts.

Publicações recentes

Ver todas no PubMed

Associações

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Comunidades

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Genetically confirmed Charcot-Marie-Tooth disease type 2A manifesting with postural tremor: a case report.
    Journal of medical case reports· 2024· PMID 39604983mais citado
  2. Roussy-L&#xe9;vy Syndrome: Pes Cavus, Tendon Areflexia, Amyotrophy, Gait Ataxia, and Upper Limb Tremor in a Patient with CMT Neuropathy.
    Tremor and other hyperkinetic movements (New York, N.Y.)· 2024· PMID 38344215mais citado
  3. Characterisation of Patients with SH3TC2 Associated Neuropathy in an Indian Cohort.
    Neurology India· 2023· PMID 37929431mais citado
  4. Roussy-L&#xe9;vy syndrome: a case of genotype-phenotype correlation.
    Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology· 2021· PMID 34232402mais citado
  5. Mutational screening of the SH3TC2 gene in Greek patients with suspected demyelinating recessive Charcot-Marie-Tooth disease reveals a varied and unusual phenotypic spectrum.
    Journal of the peripheral nervous system : JPNS· 2019· PMID 30653784mais citado

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:3115(Orphanet)
  2. OMIM OMIM:180800(OMIM)
  3. MONDO:0008392(MONDO)
  4. GARD:4741(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q3281293(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Síndrome Roussy-Lévy
Compêndio · Raras BR

Síndrome Roussy-Lévy

ORPHA:3115 · MONDO:0008392
Prevalência
Unknown
Herança
Autosomal dominant
CID-10
G60.0 · Neuropatia hereditária motora e sensorial
CID-11
Início
Childhood, Infancy
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0205713
EuropePMC
Wikidata
Papers 10a
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