A síndrome de Temtamy é uma doença neurológica genética congênita muito rara, caracterizada por agenesia/hipoplasia do corpo caloso com anormalidades de desenvolvimento, distúrbios oculares e anormalidades craniofaciais e esqueléticas variáveis.
Introdução
O que você precisa saber de cara
A síndrome de Temtamy é uma doença neurológica genética congênita muito rara, caracterizada por agenesia/hipoplasia do corpo caloso com anormalidades de desenvolvimento, distúrbios oculares e anormalidades craniofaciais e esqueléticas variáveis.
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
+ 14 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 50 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
2 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive.
In brain, may be required for corpus callosum development
Cytoplasm
Temtamy syndrome
An autosomal recessive syndrome characterized by intellectual disability, variable craniofacial dysmorphism, ocular coloboma, seizures, and brain abnormalities, including abnormalities of the corpus callosum and thalamus.
In brain, may be required for corpus callosum development
Cytoplasm
Temtamy syndrome
An autosomal recessive syndrome characterized by intellectual disability, variable craniofacial dysmorphism, ocular coloboma, seizures, and brain abnormalities, including abnormalities of the corpus callosum and thalamus.
Variantes genéticas (ClinVar)
186 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 220 variantes classificadas pelo ClinVar.
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 — Síndrome Temtamy
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
Dental Management of a Child With Temtamy Syndrome: A Case Report.
Temtamy syndrome is a rare genetic disorder that follows an autosomal recessive pattern. It is characterized by intellectual disability, developmental delays, and distinctive facial features. It may also affect the eyes, heart, and skeletal system. This case report details the dental treatment of a four-year-old male patient recently diagnosed with Temtamy syndrome. The patient complained of dental pain and presented with generalized developmental delay, intellectual disability, and hypotonia. Full dental rehabilitation under general anesthesia was planned due to behavior issues. Micrognathia and a limited mouth opening were among the challenges faced during the dental treatment. A limited mouth opening can be managed under general anesthesia, but if the child were to be treated under local anesthesia, this would further complicate the treatment. Therefore, the recommended treatment option when treating such patients is under general anesthesia to overcome these problems. This case report aims to discuss the oral features and dental management of children with Temtamy syndrome, which will aid fellow dentists in anticipating challenges faced in such cases, and further, understand the disease.
C12ORF57: a novel principal regulator of synaptic AMPA currents and excitatory neuronal homeostasis.
Excitatory neuronal homeostasis is crucial for neuronal survival, circuit function, and plasticity. Disruptions in this form of homeostasis are believed to underpin a variety of neuronal conditions including intellectual disability, epilepsy, and autism. However, the underlying genetic and molecular mechanisms maintaining this homeostasis remain poorly understood. Biallelic recurrent loss of function mutations in C12ORF57, an evolutionarily conserved X amino acid novel open reading frame, underlie Temtamy syndrome (TS)-a neurodevelopmental disorder characterized by epilepsy, dysgenesis of the corpus callosum, and severe intellectual disability. Through multiple lines of inquiry, we establish that C12ORF57/GRCC10 plays an unexpected central role in synaptic homeostatic downscaling in response to elevated activity, uncovering a novel mechanism for neuronal excitatory homeostasis. To probe these mechanisms, we developed a new knockout (KO) mouse model of the gene's murine ortholog, Grcc10 as well as cellular and in vitro assays. Grcc10 KO mice exhibit the characteristic phenotypic features seen in human TS patients, including increased epileptiform activity. Corresponding with the enhanced seizure susceptibility, hippocampal neurons in these mice exhibited significantly increased AMPA receptor expression levels and higher amplitude of miniature excitatory postsynaptic currents (mEPSCs). We further found that GRCC10/C12ORF57 modulates the activity of calcium/calmodulin dependent kinase 4 (CAMK4) and thereby regulates the expression of CREB and ARC. Our study suggests through this novel mechanism, deletion of Grcc10 disrupts the characteristic synaptic AMPA receptor downscaling that accompanies increased activity in glutamatergic neurons.
A novel pathogenic compound heterozygous variant in C12orf57 gene in a child with Temtamy syndrome presenting with overlapping phenotypic features of Kabuki-like syndrome.
Autism spectrum disorder is a major neurodevelopmental disorder. Temtamy syndrome is a rare syndromic intellectual developmental disorder that presents with global developmental delay, autism, seizures, and agenesis/dysgenesis of the corpus callosum. We report a case of a male child who presented with global developmental delay, and autism. Additional clinical features in the child were prominent eyes, long palpebral fissures with eversion of lateral third of the lower eyelid, hypoplastic nipples, and persistent fetal fingertip pads. The clinical features were in favor of Kabuki-like syndrome. MRI brain revealed corpus callosal dysgenesis, mild cerebellar para-vermian, and vermian atrophy. Trio exome sequencing has revealed a novel pathogenic compound heterozygous variant c.145A >T (p.Lys49Ter) and c.224_242del (p.Val85GlufsTer88) in exon 2 of the C12orf57 gene. This is the first case of Temtamy syndrome reported from India with additional novel phenotypic features not reported previously and broadens the phenotypic spectrum of the disorder. In addition, it expands the spectrum of pathogenic variants in the C12orf57 gene.
C12orf57 pathogenic variants: a unique cause of developmental encephalopathy in a south Indian child.
Open reading frame variants which lack stop codons such as C12orf57 variants are known to cause Temtamy syndrome, an extremely rare disorder characterized by intellectual disability, seizures, facial dysmorphism and agenesis of corpus callosum. C12orf57 was initially reported to be required for human corpus callosum development. We report the first child who is of Indian origin with developmental and epileptic encephalopathy (DEE) with a unique phenotypic evolution as focal onset reflex seizures. We performed whole exome sequencing of genomic DNA isolated from peripheral blood samples of proband and his parents. Two pathogenic compound heterozygous variants, a start loss variant (Chr12:7053285:c.1A>G) and a premature stop gain variant (Chr12:7053327:c.43C>T), involving the C12orf57 gene were identified in the proband. Our case report which details genotyping in this rare syndromic developmental encephalopathy, with no prior cases reported from India, expands the ethnic spectrum of patients.
[Ocular manifestations in Temtamy syndrome: A case report].
Publicações recentes
Dental Management of a Child With Temtamy Syndrome: A Case Report.
C12ORF57: a novel principal regulator of synaptic AMPA currents and excitatory neuronal homeostasis.
A novel pathogenic compound heterozygous variant in C12orf57 gene in a child with Temtamy syndrome presenting with overlapping phenotypic features of Kabuki-like syndrome.
[Ocular manifestations in Temtamy syndrome: A case report].
C12orf57 pathogenic variants: a unique cause of developmental encephalopathy in a south Indian child.
📚 EuropePMC7 artigos no totalmostrando 7
Dental Management of a Child With Temtamy Syndrome: A Case Report.
CureusC12ORF57: a novel principal regulator of synaptic AMPA currents and excitatory neuronal homeostasis.
bioRxiv : the preprint server for biologyA novel pathogenic compound heterozygous variant in C12orf57 gene in a child with Temtamy syndrome presenting with overlapping phenotypic features of Kabuki-like syndrome.
Brain & development[Ocular manifestations in Temtamy syndrome: A case report].
Journal francais d'ophtalmologieC12orf57 pathogenic variants: a unique cause of developmental encephalopathy in a south Indian child.
Journal of geneticsTemtamy syndrome caused by a new C12orf57 variant in a Chinese boy, including pedigree analysis and literature review.
Experimental and therapeutic medicineFurther delineation of Temtamy syndrome of corpus callosum and ocular abnormalities.
American journal of medical genetics. Part AAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Síndrome Temtamy.
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Comunidades
Grupos ativos de quem convive com esta doença aqui no Raras
Ainda não existe comunidade no Raras para Síndrome Temtamy
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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.
- Dental Management of a Child With Temtamy Syndrome: A Case Report.
- C12ORF57: a novel principal regulator of synaptic AMPA currents and excitatory neuronal homeostasis.
- A novel pathogenic compound heterozygous variant in C12orf57 gene in a child with Temtamy syndrome presenting with overlapping phenotypic features of Kabuki-like syndrome.
- C12orf57 pathogenic variants: a unique cause of developmental encephalopathy in a south Indian child.
- [Ocular manifestations in Temtamy syndrome: A case report].
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:1777(Orphanet)
- OMIM OMIM:218340(OMIM)
- MONDO:0009033(MONDO)
- GARD:5688(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q54366503(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
