A síndrome de Van den Ende-Gupta é uma condição muito rara caracterizada por olhos com abertura estreita, dedos longos e finos, dificuldade para movimentar as articulações e características físicas distintas.
Introdução
O que você precisa saber de cara
A síndrome de Van den Ende-Gupta é uma condição muito rara caracterizada por olhos com abertura estreita, dedos longos e finos, dificuldade para movimentar as articulações e características físicas distintas.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
Encontrou um erro ou informação desatualizada? Sugira uma correção →
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
+ 30 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 79 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
Encontrou um erro ou informação desatualizada? Sugira uma correção →
Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.
Probable adhesion protein, which mediates homophilic and heterophilic interactions. In contrast to SCARF1, it poorly mediates the binding and degradation of acetylated low density lipoprotein (Ac-LDL) (By similarity)
Membrane
Van den Ende-Gupta syndrome
A syndrome characterized by craniofacial and skeletal abnormalities that include blepharophimosis, a flat and wide nasal bridge, narrow and beaked nose, hypoplastic maxilla with or without cleft palate and everted lower lip, prominent ears, down-slanting eyes, arachnodactyly, and camptodactyly. Patients present congenital joint contractures that improve without intervention, and normal growth and development. Intelligence is normal. Rarely, enlarged cerebella can be present. Some patients experience respiratory problems due to laryngeal abnormalities.
Variantes genéticas (ClinVar)
373 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 29 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
1 via biológica associada 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 — Síndrome Van den Ende-Gup
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
First Prenatal Case of Genotypically and Phenotypically Overlapping Double Molecular Diagnosis of Van den Ende-Gupta and 22q11.2 Deletion Syndromes.
Multiple molecular diagnoses (MMD) involve distinct or overlapping phenotypes. They are not so rare in the field of congenital anomalies, given an overall 3.5%-8% rate. Mainly, MMD imply distinct genotypes. Exceptionally, genotypes are linked, involving a causal CNV by itself, facing a SNV for a recessive disorder resulting in a dual diagnosis. An unrelated couple was referred at 21 + 3 weeks of gestation for talipes equinovarus, cerebellar hypoplasia, clenched fists, elevated hemidiaphragm, and micrognathia. Chromosomal microarray and exome sequencing analyses were performed. Both identified a pathogenic de novo 22q11.21 deletion (22q11.2del). Fetal autopsy revealed additional features (postaxial polydactyly, facial features, and abnormal lung lobulation), atypical for 22q11.2del syndrome. At the clinician's request, exome sequencing reanalysis identified a paternally inherited SCARF2 variant, in trans to the 22q11.2del causing autosomal recessive Van den Ende-Gupta syndrome. This dual diagnosis explains the entire fetus phenotype. This is a novel case of dual diagnosis, first prenatal and second case of this ultrarare association. It reflects the crucial role of precise phenotypic description, combined with the importance of considering dual diagnosis in case of atypical clinical presentation. Finally, prenatal phenotypes remain a challenge given the paucity of available known prenatal data for most rare diseases. ClinicalTrial.gov ID: NCT05182242.
Interaction mechanism of lipid metabolism remodeling, oxidative stress, and immune response mediated by Epinephelus coioides SRECII.
Scavenger receptors (SRs), a category of pattern recognition receptors primarily expressed on the surface of myeloid cells, play pivotal roles in oxidation response and lipid metabolism. However, current research on Scavenger Receptor class F type II (SRECⅡ) revolves around Van Den Ende-Gupta Syndrome (VDEG), the potential function of SRECII, particularly in regulating lipid metabolism and oxidative stress processes, remains elusive. Herein, we elucidate that SRECII from Epinephelus coioides (EcSRECII) may modulate fatty acid oxidation and oxidative stress levels via interactions with lipoprotein particles. EcSRECII mediates the internalization of oxidized Low-Density Lipoprotein (oxLDL), thereby inducing lipid accumulation. This process upregulates the expression of genes associated with lipid synthesis and concurrently suppresses those involved in lipolysis. Additionally, EcSRECII exacerbates the production of Reactive Oxygen Species (ROS) following oxLDL exposure, evidenced by significantly heightened activities of superoxide dismutase (SOD) and glutathione peroxidase (GSH-Px), increased accumulation of malondialdehyde (MDA), and enhanced total antioxidant capacity (T-AOC). Furthermore, we reveal that EcSRECII-mediated oxLDL internalization culminates in apoptosis and necrosis in GS cells. Ultimately, we demonstrate that EcSRECII augments oxLDL-induced cellular oxidative stress and inflammatory responses, effects that are notably mitigated by EcSRECII knockdown or pretreatment with the fatty acid synthase (FAS) inhibitor C75. Collectively, our study underscores the role of EcSRECII in facilitating oxLDL internalization and subsequent lipid metabolism remodeling, thereby participating in the intricate regulation of intracellular oxidative stress and immune responses.
A rare encounter: Navigating airway challenges in Van den Ende-Gupta syndrome.
Exploring scavenger receptor class F member 2 and the importance of scavenger receptor family in prediagnostic diseases.
Scavenger Receptor Class F Member 2 (SCARF2), also known as the Type F Scavenger Receptor Family gene, encodes for Scavenger Receptor Expressed by Endothelial Cells 2 (SREC-II). This protein is a crucial component of the scavenger receptor family and is vital in protecting mammals from infectious diseases. Although research on SCARF2 is limited, mutations in this protein have been shown to cause skeletal abnormalities in both SCARF2-deficient mice and individuals with Van den Ende-Gupta syndrome (VDEGS), which is also associated with SCARF2 mutations. In contrast, other scavenger receptors have demonstrated versatile responses and have been found to aid in pathogen elimination, lipid transportation, intracellular cargo transportation, and work in tandem with various coreceptors. This review will concentrate on recent progress in comprehending SCARF2 and the functions played by members of the Scavenger Receptor Family in pre-diagnostic diseases.
Two novel variants in SCARF2 gene underlie van den Ende-Gupta syndrome.
Van den Ende-Gupta syndrome (VDEGS) (MIM#600920) is characterized by skeletal and craniofacial abnormalities that include prominent ears, downslanting palpebral fissures, blepharophimosis, hypoplastic maxilla with or without a cleft palate, a narrow and convex nasal bridge and an everted lower lip, camptodactyly and arachnodactyly. Intelligence is normal. Recent studies have reported that patients with VDEGS have pathogenic variants in the SCARF2 gene on chromosome 22q11.21. Here, we report two Turkish patients with two novel variants [c.2291_2292insC (p.Ser765LeufsTer6) and c.488G>A (p.Cys63Tyr)] in the SCARF2 gene. In silico analysis predicted that both of these novel variants were pathogenic. To the best of our knowledge, this is the first case report of this syndrome in Turkey.
Publicações recentes
A rare encounter: Navigating airway challenges in Van den Ende-Gupta syndrome.
First Prenatal Case of Genotypically and Phenotypically Overlapping Double Molecular Diagnosis of Van den Ende-Gupta and 22q11.2 Deletion Syndromes.
📖 RevisãoInteraction mechanism of lipid metabolism remodeling, oxidative stress, and immune response mediated by Epinephelus coioides SRECII.
🥈 ObservacionalExploring scavenger receptor class F member 2 and the importance of scavenger receptor family in prediagnostic diseases.
Identification of a novel variant of SCARF2 in a Jordanian family with a van den Ende-Gupta Syndrome and literature review.
📚 EuropePMC17 artigos no totalmostrando 10
A rare encounter: Navigating airway challenges in Van den Ende-Gupta syndrome.
Journal of anaesthesiology, clinical pharmacologyFirst Prenatal Case of Genotypically and Phenotypically Overlapping Double Molecular Diagnosis of Van den Ende-Gupta and 22q11.2 Deletion Syndromes.
Molecular genetics & genomic medicineInteraction mechanism of lipid metabolism remodeling, oxidative stress, and immune response mediated by Epinephelus coioides SRECII.
Free radical biology & medicineExploring scavenger receptor class F member 2 and the importance of scavenger receptor family in prediagnostic diseases.
Toxicological researchIdentification of a novel variant of SCARF2 in a Jordanian family with a van den Ende-Gupta Syndrome and literature review.
Clinical dysmorphologyTwo novel variants in SCARF2 gene underlie van den Ende-Gupta syndrome.
American journal of medical genetics. Part AFurther delineation of van den Ende-Gupta syndrome: Genetic heterogeneity and overlap with congenital heart defects and skeletal malformations syndrome.
American journal of medical genetics. Part ASimultaneous Midface Advancement and Orthognathic Surgery: A Powerful Technique for Managing Midface Hypoplasia and Malocclusion.
Plastic and reconstructive surgeryInclusion of joint laxity, recurrent patellar dislocation, and short distal ulnae as a feature of Van Den Ende-Gupta syndrome: a case report.
BMC medical geneticsDiagnosis of Van den Ende-Gupta syndrome: Approach to the Marden-Walker-like spectrum of disorders.
American journal of medical genetics. Part AAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Síndrome Van den Ende-Gup.
É de uma associação que acompanha esta doença? Fale com a gente →
Comunidades
Grupos ativos de quem convive com esta doença aqui no Raras
Ainda não existe comunidade no Raras para Síndrome Van den Ende-Gup
Pacientes, familiares e cuidadores se organizam em comunidades pra compartilhar experiências, fazer perguntas e se apoiar. Você pode ser o primeiro.
Tire suas dúvidas
Perguntas, dicas e experiências compartilhadas aqui na página
Participe da discussão
Faça login para postar dúvidas, compartilhar experiências e interagir com especialistas.
Fazer loginDoenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
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.
- First Prenatal Case of Genotypically and Phenotypically Overlapping Double Molecular Diagnosis of Van den Ende-Gupta and 22q11.2 Deletion Syndromes.
- Interaction mechanism of lipid metabolism remodeling, oxidative stress, and immune response mediated by Epinephelus coioides SRECII.
- A rare encounter: Navigating airway challenges in Van den Ende-Gupta syndrome.
- Exploring scavenger receptor class F member 2 and the importance of scavenger receptor family in prediagnostic diseases.
- Two novel variants in SCARF2 gene underlie van den Ende-Gupta syndrome.
- Identification of a novel variant of SCARF2 in a Jordanian family with a van den Ende-Gupta Syndrome and literature review.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:2460(Orphanet)
- OMIM OMIM:600920(OMIM)
- MONDO:0010959(MONDO)
- GARD:3382(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q9390616(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