A encefalopatia glicínica atípica é uma forma rara de encefalopatia glicínica (GE) que apresenta início da doença ou manifestações clínicas que diferem da EG neonatal ou infantil.
Introdução
O que você precisa saber de cara
A **encefalopatia atípica por glicina** é uma condição genética rara caracterizada por alterações na forma como o organismo processa o aminoácido glicina. Diferente de formas clássicas que surgem apenas no recém-nascido, os sinais podem iniciar em qualquer idade e costumam envolver atraso no desenvolvimento, hipotonia axial (fraqueza no tronco) e problemas visuais como atrofia óptica. Também podem ocorrer rigidez nas articulações, crises de contração muscular e dificuldades respiratórias. No Brasil, esta doença não conta com um protocolo clínico (PCDT) próprio nem remédios específicos fornecidos pelo SUS, sendo o cuidado baseado no acompanhamento dos sintomas pela rede de saúde.
A encefalopatia glicínica atípica é uma forma rara de encefalopatia glicínica (GE) que apresenta início da doença ou manifestações clínicas que diferem da EG neonatal ou infantil.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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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
+ 11 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 35 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
4 genes identificados com associação a esta condição. Padrão de herança: Unknown.
Curadoria gene-doença
fontes oficiaisNon-ketotic hyperglycinemia
Autosomal recessive disease characterized by accumulation of a large amount of glycine in body fluid and by severe neurological symptoms.
Multiple mitochondrial dysfunctions syndrome 7
An autosomal recessive disorder biochemically characterized by glycine accumulation in body fluids, including the cerebrospinal fluid, with an elevated cerebrospinal fluid/plasma glycine ratio. The broad clinical spectrum ranges from neonatal fatal glycine encephalopathy to an attenuated phenotype of developmental delay, limited verbal communication, behavioral problems, seizures and variable movement problems. Death in infancy or early childhood may occur.
Glycine encephalopathy with normal serum glycine
An autosomal recessive, severe metabolic disorder characterized by arthrogryposis multiplex congenita, joint hyperlaxity, lack of neonatal respiratory effort, axial hypotonia, hypertonia with pronounced clonus, and delayed psychomotor development. Some patients may have dysmorphic facial features and/or brain imaging abnormalities. Laboratory studies show increased CSF glycine and normal or only mildly increased serum glycine. Most patients die in infancy.
Glycine encephalopathy 2
A form of glycine encephalopathy, a metabolic disorder characterized by a high concentration of glycine in the body fluids. Affected individuals typically have severe neurological symptoms, including seizure, lethargy, and muscular hypotonia soon after birth. Most of them die within the neonatal period. Atypical cases have later disease onset and less severely affected psychomotor development.
Variantes genéticas (ClinVar)
281 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 282 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
3 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Encefalopatia atípica por glicina
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.
Publicações mais relevantes
The History and Nosology of the Glycine Disorders: A Framework for Clinicians.
Despite its simple chemical structure, glycine plays a complex role in the body. The glycine cleavage system regulates brain glycine levels and is a key one-carbon donor to folate. Its metabolism is tightly integrated with that of serine. In addition to its biochemical role, glycine functions as a neurotransmitter and neuromodulator. Primary defects in the glycine cleavage system have long been known to cause human disease with a primarily neurological phenotype, and this was labelled as 'nonketotic hyperglycinaemia' in 1968. With increasing availability of molecular testing, many additional genetic conditions became apparent, as well as non-genetic factors that cause hyperglycinaemia. There is now a much greater appreciation of the marked clinical impact of this heterogeneity. The previous terminology of 'classical' and 'atypical' nonketotic hyperglycinaemia does not adequately address these numerous genetic aetiologies, nor does it account for the phenotypic spectrum within individual genetic disorders. We provide here a clinically relevant classification of the glycine encephalopathies, based on the underlying genetic aetiology and its relation to the glycine cleavage system. Characteristic clinical and biochemical features of each condition, as well as non-genetic phenocopies that cause hyperglycinaemia, are discussed in detail. This provides a readily usable framework for clinicians when faced with a patient with elevated glycine levels.
The Application Value of Early Amplitude-Integrated Electroencephalogram in a Newborn with Nonketotic Hyperglycinemia: A Rare Case Report.
This study aimed to evaluate the application value of amplitude-integrated electroencephalogram (aEEG) findings in a newborn with nonketotic hyperglycinemia (NKH). The clinical data of a neonatal patient with NKH were retrospectively analyzed. In this study, aEEG was first used to assess brain function in NKH due to AMT gene mutations in the Chinese mainland so far. The aEEG assessment was stratified according to its background pattern, sleep-wake cycle (SWC), and seizure activity, which gave more objective and systemic results. Seizures and burst-suppression pattern were detected on the aEEG. The background belonged to discontinuous voltage, and showed discontinuity of cerebral activity in the form of the burst-suppression pattern. The classification of SWC in this record belonged to the "No SWC" category, which meant the child had severe brain damage. A typical neonatal single seizure was found. The seizure activity lasted approximately 30 seconds. However, clinical symptoms were not observed. Patients with NKH often exhibit complicated clinical phenotypes, and there is a lack of specific symptoms, especially the symptoms of encephalopathy are atypical. aEEG is helpful for the early diagnosis and treatment of seizures. It can help the doctor to carry out appropriate treatment in time. The application value of aEEG in patients with NKH was significant.
Infant With a Severe Form of GLRX5-Related Atypical Hyperglycinemia Exhibiting Novel Cardiac and Neurologic Disease Manifestations at Autopsy.
Glutaredoxin 5 (GLRX5) is a mitochondrial protein encoded by the GLRX5 gene, which is essential for cellular redox homoeostasis, lipoic acid synthesis, and iron-sulfur cluster transfer. Rare cases of pathogenic GLRX5 mutations have been associated with sideroblastic anemia and non-ketotic hyperglycinemia with progressive spasticity and cavitating leukoencephalopathy. We report an 11-month-old child, who died following aspiration, with severe cardiomyocyte mitochondrial abnormalities and cerebral white matter degeneration in the context of a homozygous GLRX5 variant (c.208A>G, p.S70G).
Case report: Unveiling genetic and phenotypic variability in Nonketotic hyperglycinemia: an atypical early onset case associated with a novel GLRX5 variant.
Nonketotic hyperglycinemia (NKH) is a rare, autosomal recessive metabolic disorder usually associated with mutations in genes AMT, GLDC or GCSH involved in the glycine cleavage complex. Other genes have been linked with less severe NKH, associated with deficiency of lipoate cofactor such as GLRX5, LIAS, BOLA3. We identified a new case of GLRX5-mediated NKH who presented at 2-month with severe developmental delay and seizures. The initial suspicion was raised by the MRI and then confirmed by glycine measurements in cerebrospinal fluid and blood. Genetic analysis revealed a previously undescribed homozygous variant in the GLRX5 gene [NM_016417.3:c.367G>C; p. (Asp123His)]. Despite medication and supportive care, he died at the age of 4 months after a sudden neurological deterioration. It was decided to limit therapeutic interventions due to the severity of the prognosis. The case was more severe than the previous GLRX5-mediated NKH described, regarding the early age at onset and the severity. Moreover, the genetic variant was located at a potentially crucial site for glutathione binding in the GLRX5 protein. This report, thereby, expands our understanding of NKH's genetic underpinnings and phenotypic variability, highlighting the crucial role of GLRX5 and other related genes in variant NKH.
[Responses to a reader's question on the article "Clinical and genetic analyses of a family with atypical nonketotic hyperglycinemia caused by compound heterozygous"].
Publicações recentes
The History and Nosology of the Glycine Disorders: A Framework for Clinicians.
The Application Value of Early Amplitude-Integrated Electroencephalogram in a Newborn with Nonketotic Hyperglycinemia: A Rare Case Report.
🥉 Relato de casoInfant With a Severe Form of GLRX5-Related Atypical Hyperglycinemia Exhibiting Novel Cardiac and Neurologic Disease Manifestations at Autopsy.
Case report: Unveiling genetic and phenotypic variability in Nonketotic hyperglycinemia: an atypical early onset case associated with a novel GLRX5 variant.
[Responses to a reader's question on the article "Clinical and genetic analyses of a family with atypical nonketotic hyperglycinemia caused by compound heterozygous"].
📚 EuropePMC1 artigos no totalmostrando 14
The History and Nosology of the Glycine Disorders: A Framework for Clinicians.
Journal of inherited metabolic diseaseThe Application Value of Early Amplitude-Integrated Electroencephalogram in a Newborn with Nonketotic Hyperglycinemia: A Rare Case Report.
AJP reportsInfant With a Severe Form of GLRX5-Related Atypical Hyperglycinemia Exhibiting Novel Cardiac and Neurologic Disease Manifestations at Autopsy.
Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology SocietyCase report: Unveiling genetic and phenotypic variability in Nonketotic hyperglycinemia: an atypical early onset case associated with a novel GLRX5 variant.
Frontiers in genetics[Responses to a reader's question on the article "Clinical and genetic analyses of a family with atypical nonketotic hyperglycinemia caused by compound heterozygous"].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatricsParkinsonian Syndrome with Frontal Lobe Involvement and Anti-Glycine Receptor Antibodies.
Brain sciencesNonketotic Hyperglycinemia: Two Case Reports and Review.
The Neurodiagnostic journalThe effect of hyperglycinemic treatment in captive-bred Vervet monkeys (Chlorocebus aethiops).
Metabolic brain diseaseDetection of MCPG metabolites in horses with atypical myopathy.
PloS oneInvestigation of neuronal auto-antibodies in children diagnosed with epileptic encephalopathy of unknown cause.
Brain & developmentRefractory chronic epilepsy associated with neuronal auto-antibodies: could perisylvian semiology be a clue?
Epileptic disorders : international epilepsy journal with videotapeMutations of the glycine cleavage system genes possibly affect the negative symptoms of schizophrenia through metabolomic profile changes.
Psychiatry and clinical neurosciences[Clinical and genetic analyses of a family with atypical nonketotic hyperglycinemia caused by compound heterozygous mutations in the GLDC gene].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatricsGlycine receptor antibody-associated epilepsy in a boy aged 4 years.
BMJ case reportsAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
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Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Encefalopatia atípica por glicina e no aconselhamento genético da família.
Doenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
Perguntas frequentes
O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu
É um subtipo raro de encefalopatia glicínica que apresenta manifestações clínicas ou momento de início diferentes das formas infantil e neonatal clássicas. Ela tem prevalência estimada de menos de 1 indivíduo em cada 1.000.000 de pessoas.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
5 publicações do grafo RarasNet (PubMed) · 7 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.
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.
Citar este verbete
Raras. (s.d.). Encefalopatia atípica por glicina. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/encefalopatia-atipica-por-glicina
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
Encefalopatia atípica por glicina
📋 Origem dos dados
Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.
- Doença rara (ontologia)
- fonte: Orphanet
- Identificador unificado
- fonte: MONDO
- Genética mendeliana
- fonte: OMIM
- Codificação WHO/SUS
- fonte: WHO ICD-10 / DATASUS
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Dado público estruturado
- fonte: Wikidata