Raras
Buscar doenças, sintomas, genes...
Vitreorretinopatia exsudativa familiar
ORPHA:891CID-10 · H35.0CID-11 · LA13.3DOENÇA RARA

A Vitreorretinopatia Exsudativa Familiar (FEVR) é uma doença rara e hereditária dos olhos que afeta o vítreo (o gel que preenche o olho) e a retina (a camada que capta a luz). Ela é caracterizada pela formação incompleta ou anormal de vasos sanguíneos na retina periférica (a parte mais externa da retina), o que pode levar a manifestações clínicas (sinais e sintomas) variadas. Essas manifestações podem ir desde a ausência total de sintomas ou pequenas alterações, até casos mais graves como o descolamento de retina, que pode levar à cegueira.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A Vitreorretinopatia Exsudativa Familiar (FEVR) é uma doença rara e hereditária dos olhos que afeta o vítreo (o gel que preenche o olho) e a retina (a camada que capta a luz). Ela é caracterizada pela formação incompleta ou anormal de vasos sanguíneos na retina periférica (a parte mais externa da retina), o que pode levar a manifestações clínicas (sinais e sintomas) variadas. Essas manifestações podem ir desde a ausência total de sintomas ou pequenas alterações, até casos mais graves como o descolamento de retina, que pode levar à cegueira.

Pesquisas ativas
1 ensaio
3 total registrados no ClinicalTrials.gov
Publicações científicas
648 artigos
Último publicado: 2026 Apr 14

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
Infancy
+ neonatal
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: H35.0
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

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

👁️
Olhos
51 sintomas
🦴
Ossos e articulações
21 sintomas
🧠
Neurológico
15 sintomas
❤️
Coração
7 sintomas
💪
Músculos
2 sintomas
😀
Face
2 sintomas

+ 43 sintomas em outras categorias

Características mais comuns

100%prev.
Vitreorretinopatia
90%prev.
Avascularização retiniana periférica
Muito frequente (99-80%)
55%prev.
Descolamento tracional da retina
Frequente (79-30%)
55%prev.
Dobra retiniana falciforme
Frequente (79-30%)
55%prev.
Neovascularização retiniana
Frequente (79-30%)
55%prev.
Morfologia anormal do disco óptico
Frequente (79-30%)
145sintomas
Muito frequente (2)
Frequente (6)
Ocasional (19)
Sem dados (118)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 145 características clínicas mais associadas, ordenadas por frequência.

VitreorretinopatiaVitreoretinopathy
Muito frequente100%
Avascularização retiniana periféricaPeripheral retinal avascularization
Muito frequente (99-80%)90%
Descolamento tracional da retinaTractional retinal detachment
Frequente (79-30%)55%
Dobra retiniana falciformeFalciform retinal fold
Frequente (79-30%)55%
Neovascularização retinianaRetinal neovascularization
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico648PubMed
Últimos 10 anos200publicações
Pico202557 papers
Linha do tempo
2026Hoje · 2026🧪 2005Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

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

7 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant, Autosomal recessive, X-linked recessive.

FZD4Frizzled-4Disease-causing germline mutation(s) inModerado
FUNÇÃO

Receptor for Wnt proteins (PubMed:30135577). Most frizzled receptors are coupled to the beta-catenin (CTNNB1) canonical signaling pathway, which leads to the activation of disheveled proteins, inhibition of GSK-3 kinase, nuclear accumulation of beta-catenin (CTNNB1) and activation of Wnt target genes (PubMed:30135577). Plays a critical role in retinal vascularization by acting as a receptor for Wnt proteins and norrin (NDP) (By similarity). In retina, it can be activated by Wnt protein-binding a

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (8)
Ca2+ pathwayAsymmetric localization of PCP proteinsWNT5A-dependent internalization of FZD4Clathrin-mediated endocytosisCargo recognition for clathrin-mediated endocytosis
MECANISMO DE DOENÇA

Vitreoretinopathy, exudative 1

An autosomal dominant disorder of the retinal vasculature characterized by an abrupt cessation of growth of peripheral capillaries, leading to an avascular peripheral retina. This may lead to compensatory retinal neovascularization, which is thought to be induced by hypoxia from the initial avascular insult. New vessels are prone to leakage and rupture causing exudates and bleeding, followed by scarring, retinal detachment and blindness. Clinical features can be highly variable, even within the same family. Patients with mild forms of the disease are asymptomatic, and their only disease related abnormality is an arc of avascular retina in the extreme temporal periphery. In many ways the disease resembles retinopathy of prematurity but there is no evidence of prematurity or small birth weight in the patient history.

EXPRESSÃO TECIDUAL(Ubíquo)
Tecido adiposo
153.3 TPM
Adipose Visceral Omentum
118.7 TPM
Mama
83.3 TPM
Pulmão
72.9 TPM
Artéria coronária
62.9 TPM
OUTRAS DOENÇAS (4)
exudative vitreoretinopathy 1persistent hyperplastic primary vitreousexudative vitreoretinopathyretinopathy of prematurity
HGNC:4042UniProt:Q9ULV1
DYNC1H1Cytoplasmic dynein 1 heavy chain 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Cytoplasmic dynein 1 acts as a motor for the intracellular retrograde motility of vesicles and organelles along microtubules. Dynein has ATPase activity; the force-producing power stroke is thought to occur on release of ADP. Plays a role in mitotic spindle assembly and metaphase plate congression (PubMed:27462074)

LOCALIZAÇÃO

Cytoplasm, cytoskeleton

VIAS BIOLÓGICAS (10)
AggrephagyAmplification of signal from unattached kinetochores via a MAD2 inhibitory signalRHO GTPases Activate ForminsMitotic PrometaphaseEML4 and NUDC in mitotic spindle formation
MECANISMO DE DOENÇA

Charcot-Marie-Tooth disease, axonal, type 2O

An axonal 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). Neuropathies of the CMT2 group are characterized by signs of axonal degeneration in the absence of obvious myelin alterations, normal or slightly reduced nerve conduction velocities, and progressive distal muscle weakness and atrophy.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
147.9 TPM
Cérebro - Hemisfério cerebelar
140.0 TPM
Cerebelo
129.1 TPM
Nervo tibial
80.3 TPM
Artéria tibial
78.4 TPM
OUTRAS DOENÇAS (4)
autosomal dominant childhood-onset proximal spinal muscular atrophy without contracturesCharcot-Marie-Tooth disease axonal type 2Ointellectual disability, autosomal dominant 13autosomal dominant non-syndromic intellectual disability
HGNC:2961UniProt:Q14204
TSPAN12Tetraspanin-12Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Regulator of cell surface receptor signal transduction. Plays a central role in retinal vascularization by regulating norrin (NDP) signal transduction. Acts in concert with norrin (NDP) to promote FZD4 multimerization and subsequent activation of FZD4, leading to promote accumulation of beta-catenin (CTNNB1) and stimulate LEF/TCF-mediated transcriptional programs. Suprisingly, it only activates the norrin (NDP)-dependent activation of FZD4, while it does not activate the Wnt-dependent activation

LOCALIZAÇÃO

Cell membrane

MECANISMO DE DOENÇA

Vitreoretinopathy, exudative 5

An autosomal dominant form of exudative vitreoretinopathy, a disorder of the retinal vasculature characterized by an abrupt cessation of growth of peripheral capillaries, leading to an avascular peripheral retina. This may lead to compensatory retinal neovascularization, which is thought to be induced by hypoxia from the initial avascular insult. New vessels are prone to leakage and rupture causing exudates and bleeding, followed by scarring, retinal detachment and blindness. Clinical features can be highly variable, even within the same family. Patients with mild forms of the disease are asymptomatic, and their only disease related abnormality is an arc of avascular retina in the extreme temporal periphery.

EXPRESSÃO TECIDUAL(Ubíquo)
Glândula adrenal
48.0 TPM
Tireoide
36.6 TPM
Rim - Medula
31.7 TPM
Pulmão
29.9 TPM
Artéria tibial
28.1 TPM
INTERAÇÕES PROTEICAS (5)
OUTRAS DOENÇAS (2)
exudative vitreoretinopathy 5exudative vitreoretinopathy
HGNC:21641UniProt:O95859
CTNNB1Catenin beta-1Disease-causing germline mutation(s) (loss of function) inAltamente restrito
FUNÇÃO

Key downstream component of the canonical Wnt signaling pathway (PubMed:17524503, PubMed:18077326, PubMed:18086858, PubMed:18957423, PubMed:21262353, PubMed:22155184, PubMed:22647378, PubMed:22699938). In the absence of Wnt, forms a complex with AXIN1, AXIN2, APC, CSNK1A1 and GSK3B that promotes phosphorylation on N-terminal Ser and Thr residues and ubiquitination of CTNNB1 via BTRC and its subsequent degradation by the proteasome (PubMed:17524503, PubMed:18077326, PubMed:18086858, PubMed:189574

LOCALIZAÇÃO

CytoplasmNucleusCytoplasm, cytoskeletonCell junction, adherens junctionCell junctionCell membraneCytoplasm, cytoskeleton, microtubule organizing center, centrosomeCytoplasm, cytoskeleton, spindle poleSynapseCytoplasm, cytoskeleton, cilium basal body

VIAS BIOLÓGICAS (10)
Formation of the nephric ductSpecification of the neural plate borderSynthesis, secretion, and inactivation of Glucagon-like Peptide-1 (GLP-1)TCF dependent signaling in response to WNTTranscriptional Regulation by VENTX
MECANISMO DE DOENÇA

Colorectal cancer

A complex disease characterized by malignant lesions arising from the inner wall of the large intestine (the colon) and the rectum. Genetic alterations are often associated with progression from premalignant lesion (adenoma) to invasive adenocarcinoma. Risk factors for cancer of the colon and rectum include colon polyps, long-standing ulcerative colitis, and genetic family history.

EXPRESSÃO TECIDUAL(Ubíquo)
Cervix Endocervix
297.5 TPM
Cervix Ectocervix
257.8 TPM
Artéria tibial
233.5 TPM
Ovário
201.9 TPM
Cérebro - Hemisfério cerebelar
201.3 TPM
OUTRAS DOENÇAS (17)
hepatocellular carcinomasevere intellectual disability-progressive spastic diplegia syndromeovarian cancerpilomatrixoma
HGNC:2514UniProt:P35222
NDPNorrinDisease-causing germline mutation(s) inRestrito
FUNÇÃO

Activates the canonical Wnt signaling pathway through FZD4 and LRP5 coreceptor. Plays a central role in retinal vascularization by acting as a ligand for FZD4 that signals via stabilizing beta-catenin (CTNNB1) and activating LEF/TCF-mediated transcriptional programs. Acts in concert with TSPAN12 to activate FZD4 independently of the Wnt-dependent activation of FZD4, suggesting the existence of a Wnt-independent signaling that also promote accumulation the beta-catenin (CTNNB1). May be involved i

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Interconversion of nucleotide di- and triphosphates
MECANISMO DE DOENÇA

Norrie disease

Recessive disorder characterized by very early childhood blindness due to degenerative and proliferative changes of the neuroretina. Approximately 50% of patients show some form of progressive mental disorder, often with psychotic features, and about one-third of patients develop sensorineural deafness in the second decade. In addition, some patients have more complex phenotypes, including growth failure and seizure.

EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
40.0 TPM
Ovário
39.0 TPM
Cerebelo
34.9 TPM
Brain Caudate basal ganglia
30.1 TPM
Brain Frontal Cortex BA9
27.6 TPM
OUTRAS DOENÇAS (6)
Norrie diseaseexudative vitreoretinopathy 2, X-linkedpersistent hyperplastic primary vitreousCoats disease
HGNC:7678UniProt:Q00604
LRP5Low-density lipoprotein receptor-related protein 5Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Acts as a coreceptor with members of the frizzled family of seven-transmembrane spanning receptors to transduce signal by Wnt proteins (PubMed:11336703, PubMed:11448771, PubMed:11719191, PubMed:15778503, PubMed:15908424, PubMed:16252235). Activates the canonical Wnt signaling pathway that controls cell fate determination and self-renewal during embryonic development and adult tissue regeneration (PubMed:11336703, PubMed:11719191). In particular, may play an important role in the development of t

LOCALIZAÇÃO

MembraneEndoplasmic reticulum

VIAS BIOLÓGICAS (1)
Negative regulation of TCF-dependent signaling by WNT ligand antagonists
MECANISMO DE DOENÇA

Vitreoretinopathy, exudative 1

An autosomal dominant disorder of the retinal vasculature characterized by an abrupt cessation of growth of peripheral capillaries, leading to an avascular peripheral retina. This may lead to compensatory retinal neovascularization, which is thought to be induced by hypoxia from the initial avascular insult. New vessels are prone to leakage and rupture causing exudates and bleeding, followed by scarring, retinal detachment and blindness. Clinical features can be highly variable, even within the same family. Patients with mild forms of the disease are asymptomatic, and their only disease related abnormality is an arc of avascular retina in the extreme temporal periphery. In many ways the disease resembles retinopathy of prematurity but there is no evidence of prematurity or small birth weight in the patient history.

EXPRESSÃO TECIDUAL(Ubíquo)
Aorta
83.2 TPM
Artéria tibial
67.8 TPM
Glândula salivar
64.2 TPM
Útero
56.7 TPM
Tireoide
51.4 TPM
OUTRAS DOENÇAS (12)
polycystic liver disease 4 with or without kidney cystsobsolete bone mineral density quantitative trait locus 1autosomal dominant osteosclerosis, Worth typeosteoporosis-pseudoglioma syndrome
HGNC:6697UniProt:O75197
ZNF408Zinc finger protein 408Disease-causing germline mutation(s) inTolerante
FUNÇÃO

May be involved in transcriptional regulation

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Vitreoretinopathy, exudative 6

An autosomal dominant form of exudative vitreoretinopathy, a form of exudative vitreoretinopathy, a disorder of the retinal vasculature characterized by an abrupt cessation of growth of peripheral capillaries, leading to an avascular peripheral retina. This may lead to compensatory retinal neovascularization, which is thought to be induced by hypoxia from the initial avascular insult. New vessels are prone to leakage and rupture causing exudates and bleeding, followed by scarring, retinal detachment and blindness. Clinical features can be highly variable, even within the same family. Patients with mild forms of the disease are asymptomatic, and their only disease related abnormality is an arc of avascular retina in the extreme temporal periphery.

EXPRESSÃO TECIDUAL(Ubíquo)
Skin Sun Exposed Lower leg
17.9 TPM
Útero
17.1 TPM
Skin Not Sun Exposed Suprapubic
15.7 TPM
Bladder
15.6 TPM
Fallopian Tube
15.5 TPM
OUTRAS DOENÇAS (4)
exudative vitreoretinopathy 6retinitis pigmentosa 72exudative vitreoretinopathyretinitis pigmentosa
HGNC:20041UniProt:Q9H9D4

Variantes genéticas (ClinVar)

1,101 variantes patogênicas registradas no ClinVar.

🧬 FZD4: NM_012193.4(FZD4):c.568C>T (p.Gln190Ter) ()
🧬 FZD4: NM_012193.4(FZD4):c.1328del (p.Leu443fs) ()
🧬 FZD4: NM_012193.4(FZD4):c.1007del (p.Gly336fs) ()
🧬 FZD4: NM_012193.4(FZD4):c.14del (p.Gly5fs) ()
🧬 FZD4: NM_012193.4(FZD4):c.470T>A (p.Met157Lys) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 17 variantes classificadas pelo ClinVar.

11
6
Patogênica (64.7%)
VUS (35.3%)
VARIANTES MAIS SIGNIFICATIVAS
LRP5: NM_002335.4(LRP5):c.1042C>T (p.Arg348Trp) [Likely pathogenic]
LRP5: NC_000011.9:g.(?_68080076)_(68216744_?)del [Pathogenic]
LRP5: NM_002335.4(LRP5):c.3763+2T>C [Conflicting classifications of pathogenicity]
ZNF408: NM_024741.3(ZNF408):c.1697T>A (p.Leu566His) [Pathogenic]
FZD4: NM_012193.4(FZD4):c.349T>C (p.Cys117Arg) [Likely pathogenic]

Vias biológicas (Reactome)

60 vias biológicas associadas aos genes desta condição.

Class B/2 (Secretin family receptors) Ca2+ pathway Asymmetric localization of PCP proteins Regulation of FZD by ubiquitination WNT5A-dependent internalization of FZD4 Signaling by RNF43 mutants Cargo recognition for clathrin-mediated endocytosis Clathrin-mediated endocytosis Amplification of signal from unattached kinetochores via a MAD2 inhibitory signal MHC class II antigen presentation Separation of Sister Chromatids Resolution of Sister Chromatid Cohesion Regulation of PLK1 Activity at G2/M Transition HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand Loss of Nlp from mitotic centrosomes Recruitment of mitotic centrosome proteins and complexes Loss of proteins required for interphase microtubule organization from the centrosome Recruitment of NuMA to mitotic centrosomes Anchoring of the basal body to the plasma membrane RHO GTPases Activate Formins Neutrophil degranulation COPI-mediated anterograde transport COPI-independent Golgi-to-ER retrograde traffic Mitotic Prometaphase AURKA Activation by TPX2 HCMV Early Events Aggrephagy EML4 and NUDC in mitotic spindle formation Degradation of beta-catenin by the destruction complex Beta-catenin phosphorylation cascade TCF dependent signaling in response to WNT Formation of the beta-catenin:TCF transactivating complex LRR FLII-interacting protein 1 (LRRFIP1) activates type I IFN production Apoptotic cleavage of cell adhesion proteins Deactivation of the beta-catenin transactivating complex Synthesis, secretion, and inactivation of Glucagon-like Peptide-1 (GLP-1) Adherens junctions interactions Binding of TCF/LEF:CTNNB1 to target gene promoters Disassembly of the destruction complex and recruitment of AXIN to the membrane VEGFR2 mediated vascular permeability Myogenesis Signaling by GSK3beta mutants CTNNB1 S33 mutants aren't phosphorylated CTNNB1 S37 mutants aren't phosphorylated CTNNB1 S45 mutants aren't phosphorylated CTNNB1 T41 mutants aren't phosphorylated RHO GTPases activate IQGAPs Transcriptional Regulation by VENTX InlA-mediated entry of Listeria monocytogenes into host cells RUNX3 regulates WNT signaling Cardiogenesis Germ layer formation at gastrulation Regulation of CDH11 function Regulation of CDH19 Expression and Function Regulation of CDH1 Function Degradation of CDH1 Regulation of CDH1 posttranslational processing and trafficking to plasma membrane Interconversion of nucleotide di- and triphosphates Negative regulation of TCF-dependent signaling by WNT ligand antagonists Signaling by LRP5 mutants

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
Aprovado1
2Fase 21
·Pré-clínico1
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 3 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Vitreorretinopatia exsudativa familiar

🗺️

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

🟢 Recrutando agora

1 pesquisa recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

3 ensaios clínicos encontrados, 1 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
🧪 Está conduzindo uma pesquisa?
Divulgue para pacientes e familiares que acompanham esta doença.
Divulgar pesquisa →

Publicações mais relevantes

Timeline de publicações
435 papers (10 anos)
#1

KIF11 prevents retinal endothelial ferroptosis in familial exudative vitreoretinopathy by inhibiting phosphorylation-driven PRDX1 phase separation.

Nature communications2026 Mar 24

Familial exudative vitreoretinopathy is a hereditary disorder predominantly affecting infants and young children, often leading to severe vision loss. Approximately 40% of patients carry mutations in Norrin/β-catenin pathway genes. Nevertheless, the downstream pathogenic mechanisms remain unclear. Here, by using bulk RNA sequencing and single-cell RNA sequencing analyses, we identify KIF11 as a key downstream effector in retinal endothelial cells. Lentivirus-mediated KIF11 overexpression partially restores vascular defects in endothelial cell-specific Ctnnb1 knockout mice. Functional and multi-omics studies reveal that β-catenin/KIF11 deficiency induces autophagy-accompanied ferroptosis. Mechanistically, KIF11 binds PRDX1, and the disrupted β-catenin/KIF11 axis releases the competitive restraint of KIF11 on Src-mediated PRDX1 phosphorylation, triggering subsequent liquid-liquid phase separation. Treatment with the ferroptosis inhibitor ferrostatin-1 or lentiviral overexpression of non-phosphorylatable PRDX1 partially rescues vascular defects in familial exudative vitreoretinopathy-associated mice. Overall, we elucidate a β-catenin/KIF11/PRDX1 axis-dependent ferroptosis mechanism in familial exudative vitreoretinopathy, highlighting ferroptosis-targeting and antioxidant strategies as potential therapies.

#2

An induced pluripotent stem cell line (SJTUXHi003-A) derived from a patient with copy number variation in the gene LRP5 causing familial exudative vitreoretinopathy.

Stem cell research2026 Feb

Familial exudative vitreoretinopathy (FEVR) is an inherited disease of retinal vascular development, and mutations in the LRP5 gene are associated with this disease. In this study, we generated a new induced pluripotent stem cell (iPSC) line, SJTUXHi003-A, from a patient with a novel copy number variation (CNV), exons 19-21 deletion in LRP5. This iPSC line exhibited a normal male karyotype with positive pluripotency markers, and could differentiate into three germ layers in vitro, providing a valuable model for studying the pathological mechanism of FEVR in vitro.

#3

Novel nonsense variant of KIF11 in a patient with MCLMR.

Human genome variation2026 Mar 02

Microcephaly with or without chorioretinopathy, lymphedema or mental retardation is a rare KIF11-related disorder. Here we report the case of a patient with microcephaly, lymphedema, nystagmus and familial exudative vitreoretinopathy carrying a novel de novo KIF11 nonsense variant (NM_004523.4:p.Glu123Ter), which is considered pathogenic. This case expands the phenotypic range of KIF11 pathogenic variants and highlights the importance of early ophthalmological evaluation, genetic counseling and family assessment.

#4

A novel variant p.Y250C of FZD4 influences Norrine/β-catenin signaling pathway that associates with familial exudative vitreoretinopathy (FEVR).

Scientific reports2026 Jan 12

Frizzled-4 (FZD4) gene mutation is a known mechanism of familial exudative vitreoretinopathy (FEVR). To establish the pathogenicity of the novel FZD4 mutation c.A749G, functional studies are needed to connect this mutation to the patient's FEVR phenotypes. Fluorescence microscopy and co-immunoprecipitation (Co-IP) techniques were employed to determine the effect of FZD4 mutation on sub-cellular localization and interaction with partners. The activity of Norrin (NDP)/β-catenin pathway was assessed through the western blot and luciferase assays. Western blot was performed to evaluate the spatial and temporal expressions of the Fzd4 across various mouse tissues. The mutated [c.A749G (p.Y250C)] forms of Frizzled 4 (FZD4) constructs were successfully conducted. Wild-type FZD4 predominantly located in the cytoplasm and plasma membrane, while the mutant exhibited a tendency to aggregate at nuclear membrane and within the nucleus. Co-IP revealed preserved mutant FZD4-LRP5 (low-density lipoprotein receptor-related protein 5) binding, suggesting the formation of receptor complex was likely unaffected by this mutation. Overexpression of mutant FZD4 and NDP or activation with agonist R-Spordin 1 (RSPO1) reduced downstream signaling proteins, including phosphorylated β-catenin (p-β-catenin), and vascular endothelial growth factor (VEGF-A). β-catenin report activity was significant lower in mutant group (P < 0.05), aligning with attenuated pathway activation. Fzd4 exhibited broad tissue expression, and it was notably present during the early developmental stages of retinal and ocular formation in mice. The novel missense mutation c.A749G in the FZD4 gene may impair the Norrin/β-catenin signaling pathway and alter subcellular localization, thereby driving FEVR pathogenesis.

#5

Comparative analysis of activation of macrophages/microglia in diabetic retinopathy and Familial Exudative Vitreoretinopathy.

Biochemistry and biophysics reports2026 Mar

Familial Exudative Vitreoretinopathy (FEVR) and Diabetic Retinopathy (DR) are two prominent retinal diseases. The role of macrophages/microglia in the vascular dynamics of FEVR and DR is unknown and thus addressed in this study. FZD4 knockout mouse, a model for FEVR in human characterized by genetic mutations affecting angiogenesis, exhibited reduced b-wave amplitudes and decreased vascular density, replicating human FEVR symptoms. Conversely, STZ-treated C57/BL6 mouse developed heightened fasting glucose levels, reduced insulin content, and increased retinal vasculature, aligning with DR features. Further analysis revealed significant differences in macrophage/microglia populations between the two diseases. In DR, a marked increase in both number and M2-like polarization of retinal macrophages/microglia was observed, contrasting with FEVR. Moreover, DR induced substantial proinflammatory differentiation of macrophages/microglia, evidenced by elevated cytokines such as IL-1β, TNF-α, and IFNɣ. Both conditions significantly upregulated Ang-1 and IL-10, with a more pronounced IL-10 increase in DR, suggesting a more active role in tissue and vessel remodeling. Notably, DR induced higher levels of anti-inflammatory factors like bFGF, TIMP-1, TGFβ1, and VEGF-A compared to FEVR, suggesting a balance of inflammation initiation, progression and resolution. These findings highlight the distinct roles of macrophages/microglia in FEVR and DR, providing insights into their contributions to disease pathogenesis and potential therapeutic strategies through reprogramming macrophages/microglia.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC405 artigos no totalmostrando 200

2026

KIF11 prevents retinal endothelial ferroptosis in familial exudative vitreoretinopathy by inhibiting phosphorylation-driven PRDX1 phase separation.

Nature communications
2026

Dragging the macula: a comet‑like falciform retinal fold in NDP‑related familial exudative vitreoretinopathy in a child.

Eye (London, England)
2026

Novel nonsense variant of KIF11 in a patient with MCLMR.

Human genome variation
2026

Misdiagnosis in Referrals for Advanced Retinopathy of Prematurity.

Ophthalmology. Retina
2026

A novel variant p.Y250C of FZD4 influences Norrine/β-catenin signaling pathway that associates with familial exudative vitreoretinopathy (FEVR).

Scientific reports
2026

An induced pluripotent stem cell line (SJTUXHi003-A) derived from a patient with copy number variation in the gene LRP5 causing familial exudative vitreoretinopathy.

Stem cell research
2025

Early Onset High Myopia and Severe Anisometropia Associated With Familial Exudative Vitreoretinopathy of Irregular Dominant Inheritance in 11 Chinese Families: Analysis of Refraction Features and Pathogenic Variations.

Genetics research
2026

Comparative analysis of activation of macrophages/microglia in diabetic retinopathy and Familial Exudative Vitreoretinopathy.

Biochemistry and biophysics reports
2026

Sphingosine-1-phosphate receptor 2 inhibition ameliorates familial exudative vitreoretinopathy models.

The Journal of biological chemistry
2025

Encircling scleral buckling as sole therapy for retinal detachment and neovascularization in familial exudative vitreo retinopathy.

European journal of ophthalmology
2026

Analysis of familial exudative vitreoretinopathy (FEVR) cases in the UK 100 000 genomes project increases diagnostic rate and implicates heterozygous CTNND1 mutations in FEVR.

Journal of medical genetics
2025

A Novel Frameshift Variant in KIF11 Causes Autosomal Dominant Familial Exudative Vitreoretinopathy in a Chinese Family.

Genetic testing and molecular biomarkers
2026

A novel TSPAN12 mutation causing retinitis pigmentosa-like appearance of familial exudative vitreoretinopathy.

Ophthalmic genetics
2025

Late Reactivation of Retinopathy in a Treatment-Naive Female Adult Patient With History of Prematurity and ZNF408 Mutation.

Journal of vitreoretinal diseases
2025

KIF11 variants in familial exudative vitreoretinopathy leading to mTORC1 overactivation and impaired cell cycle progression.

Human genomics
2025

Analysis of full-term neonatal eye disease screening results and trends from 2016 to 2023.

Scientific reports
2025

Schisis-Like Presentations of Familial Exudative Vitreoretinopathy Caused by FZD4 Mutations.

Journal of vitreoretinal diseases
2025

Feasibility of Multimodal Deep Learning for Automated Staging of Familial Exudative Vitreoretinopathy Using Color Fundus Photographs and Fluorescein Angiography.

Diagnostics (Basel, Switzerland)
2025

Refractive Error Correction With Glasses in Congenital Ocular Fundus Anomalies: A Retrospective Series of 18 Children With Different Disease Entities Followed Up for More Than 10 Years.

Cureus
2025

A novel variant in the FZD4 gene leading to familial exudative vitreoretinopathy: A case report and literature review.

Medicine
2025

Severe Exudative Vitreoretinopathy Secondary to Homozygous PCDH12 Mutations.

Retinal cases &amp; brief reports
2026

EFFICACY AND VASCULAR REDEVELOPMENT OF ANTI-VASCULAR ENDOTHELIAL GROWTH FACTOR THERAPY FOR STAGE 2 EARLY-DIAGNOSED FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retina (Philadelphia, Pa.)
2025

5' UTR variant in the NDP gene leads to incorrect splicing and familial exudative vitreoretinopathy.

Orphanet journal of rare diseases
2025

Rigid gas permeable contact lens correction for young children with special refractive errors.

Clinical &amp; experimental optometry
2026

Unveiling Endothelial Cell Expression Profiles in FEVR: Identification of Key Genes Associated With Pathological Neovascularisation in a FZD4M105V Mouse Model.

Clinical &amp; experimental ophthalmology
2025

Five novel pathogenic FZD4 variants identified in familial exudative vitreoretinopathy.

Advances in ophthalmology practice and research
2026

KIF11-related MCLMR presenting with FEVR-like retinopathy: first report in an Indian child.

Ophthalmic genetics
2025

A genotype to phenotype relationship of exudative vitreoretinopathy in Loeys-Dietz syndrome due to a pathogenic variant in TGFBR2.

Ophthalmic genetics
2026

Clinical Characteristics and Long-term Visual Prognosis of Familial Exudative Vitreoretinopathy.

American journal of ophthalmology
2026

Cellular-Level Assessment of Macular Development in Patients With Familial Exudative Vitreoretinopathy Using Multimodal Imaging: A Prospective Cohort Study.

American journal of ophthalmology
2026

EXCISION OF EXTENSIVE SUBRETINAL FIBROSIS ASSOCIATED WITH RHEGMATOGENOUS RETINAL DETACHMENTS IN PATIENTS WITH FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retina (Philadelphia, Pa.)
2025

Familial Exudative Vitreoretinopathy in a Patient with Jacobsen Syndrome: A Case Report.

Korean journal of ophthalmology : KJO
2025

Bilateral tractional retinal detachments complicating hemolytic-uremic syndrome.

American journal of ophthalmology case reports
2025

Retinal parameter analysis and diagnostic potentail exploration in familial exudative vitreoretinopathy using ultra-widefield fundus photography.

International journal of retina and vitreous
2025

Familial Exudative Vitreoretinopathy-Like Retinal Findings in Adams-Oliver Syndrome Type 2.

Clinical &amp; experimental ophthalmology
2025

An analysis of LRP5 gene frequencies in infants with familial exudative vitreoretinopathy in Chongqing and Urumqi.

Medicine
2025

Diagnostic Accuracy of AI Models in Detecting Different Inherited Retinal Diseases: A Systematic Review and Meta-Analysis.

Translational vision science &amp; technology
2025

The MDM2-p53 axis regulates norrin/frizzled4 signaling and blood-CNS barrier function.

Science signaling
2025

The diagnostic value of ultra-widefield fundus imaging technology in early familial exudative vitreoretinopathy.

Ophthalmic genetics
2025

Distinct structural mechanisms of LGR4 modulation by Norrin and RSPOs in Wnt/β-catenin signaling.

Nature communications
2025

Phenotyping and genotyping FEVR: Molecular genetics, clinical and imaging features, and therapeutics.

Progress in retinal and eye research
2025

Genotypic Distribution and Clinical Correlations in Familial Exudative Vitreoretinopathy: A Single-Center Study.

Ophthalmology science
2025

Novel KIF11 Mutation Associated with Microcephaly, Chorioretinopathy and Impaired Intellectual Development: 20 Years of Follow-Up.

Children (Basel, Switzerland)
2025

A case of congenital heart defects and familial exudative vitreoretinopathy caused by activation of a cryptic splice donor in NOTCH1.

BMC medical genomics
2025

Retinal vascularization from a new angle: A systematic review of temporal retinal vessel angles in familial exudative vitreoretinopathy.

Retina (Philadelphia, Pa.)
2025

DYRK1A syndrome presenting with a familial exudative vitreoretinopathy (FEVR)-like retinovascular phenotype.

Ophthalmic genetics
2025

Temporal Retinal Vessel Angle as a Biomarker for Familial Exudative Vitreoretinopathy.

Retina (Philadelphia, Pa.)
2025

Ultra-Widefield Swept-Source OCTA Findings in Coats Plus Syndrome.

Ophthalmic surgery, lasers &amp; imaging retina
2025

Rare pediatric retinal diseases: A review.

Indian journal of ophthalmology
2025

Impact of neonatal family screening in early-onset management of TSPAN 12 positive Familial Exudative Vitreoretinopathy- a case report.

Retinal cases &amp; brief reports
2025

Macular Morphological Changes in Familial Exudative Vitreoretinopathy with Macular Traction on OCT.

Case reports in ophthalmology
2025

Two Cases of Rubinstein-Taybi Syndrome With Retinal Detachment.

Cureus
2025

Vessels Spanning Avascular Retina in Familial Exudative Vitreoretinopathy.

Ophthalmology. Retina
2025

Fluorescein Angiography May Predict Surgical Outcomes of Tractional Retinal Detachment in Familial Exudative Vitreoretinopathy.

Ophthalmology. Retina
2025

Familial exudative vitreoretinopathy caused by CTNNB1 gene de novo mutation in a Chinese family: a case report.

BMC pediatrics
2025

Underlying Disease in Atypical Retinopathy of Prematurity.

American journal of ophthalmology
2025

Pediatric Eye Screening: Current Standards and Gaps in Care.

Ophthalmic surgery, lasers &amp; imaging retina
2025

INTRAOPERATIVE 3-DIMENSIONAL FLUORESCEIN ANGIOGRAPHY-GUIDED PARS PLANA VITRECTOMY FOR THE TREATMENT OF MULTIPLE RETINAL DISEASES.

Retina (Philadelphia, Pa.)
2025

Gene Variant Spectrum in Probands With Familial Exudative Vitreoretinopathy Using an Expanded Panel.

Investigative ophthalmology &amp; visual science
2025

Genotype-Phenotype Spectrum of eyeGENE Patients With Familial Exudative Vitreoretinopathy: Novel Variants in Norrin/β-Catenin Signaling Pathway Genes.

Investigative ophthalmology &amp; visual science
2025

Congenital retinal folds.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2025

Hereditary Vitreoretinopathies: Molecular Diagnosis, Clinical Presentation and Management.

Clinical &amp; experimental ophthalmology
2025

Utility of Fluorescein Angiography for Early Detection of Familial Exudative Vitreoretinopathy in Neurodevelopmental Disorder With Spastic Diplegia and Visual Defects Due to CTNNB1 Variants.

Journal of pediatric ophthalmology and strabismus
2024

The Evaluation of Ocular Posterior Segment Findings in 5527 Term Infants Using Smartphone-Based Fundus Imaging.

Journal of ophthalmology
2025

LONG-TERM FUNCTIONAL AND CLINICAL OUTCOMES IN FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retina (Philadelphia, Pa.)
2026

Optic Disc Drusen and Familial Exudative Vitreoretinopathy Phenotype: A Multimodal Imaging Perspective on a Rare Association.

Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society
2025

Highly asymmetric early presentation of FEVR requiring enucleation.

Ophthalmic genetics
2025

Angiographic features of pediatric stage 4 familial exudative vitreoretinopathy with radial retinal folds.

Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie
2025

SURGICAL OUTCOMES OF TRACTIONAL MACULOPATHY ASSOCIATED WITH FAMILIAL EXUDATIVE VITREORETINOPATHY IN CHILDREN.

Retina (Philadelphia, Pa.)
2025

Clinical Profiles of Retinal Vasoproliferative Tumors.

Journal of vitreoretinal diseases
2025

Improvement in Cystoid Macular Edema Secondary to Systemic Bevacizumab in a Patient With Coats Plus Syndrome.

Journal of vitreoretinal diseases
2024

Midperipheral Microvascular Defects and Their Associations With Vitreoretinal Abnormalities in Early-Stage Familial Exudative Vitreoretinopathy.

Investigative ophthalmology &amp; visual science
2024

Dysfunction of Calcyphosine-Like gene impairs retinal angiogenesis through the MYC axis and is associated with familial exudative vitreoretinopathy.

eLife
2024

FEVR combined with macular heterotopia in children presenting as pseudo-exotropia: a case report and literature review.

Frontiers in medicine
2025

Angiographic Characteristics in Mild Familial Exudative Vitreoretinopathy with Genetically Confirmed Autosomal Dominant Inheritance.

Ophthalmology. Retina
2025

PRETERM FAMILIAL EXUDATIVE VITREORETINOPATHY: A NOVEL NONSENSE LRP5 MUTATION.

Retinal cases &amp; brief reports
2024

ANATOMIC OUTCOMES OF LENS-SPARING VITRECTOMY FOR STAGE 3 OR 4 FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retina (Philadelphia, Pa.)
2024

Aggressive Onset of a Progressive FEVR Phenotype in a Child With Novel Mutations in LRP5 and TSPAN12.

Journal of vitreoretinal diseases
2024

Loss of ZNF408 attenuates STING-mediated immune surveillance in breast carcinogenesis.

iScience
2024

The Red Reflex Test and Leukocoria in Childhood.

Acta medica portuguesa
2024

Cyanoacrylate glue for iatrogenic retinal breaks during vitrectomy in stage 5 familial exudative vitreoretinopathy.

Frontiers in medicine
2024

Revision of Initial Referral Diagnosis after Genotypic Confirmation of Familial Exudative Vitreoretinopathy.

Ophthalmology. Retina
2024

Familial Exudative Vitreoretinopathy With and Without Pathogenic Variants of Norrin/β-Catenin Signaling Genes.

Ophthalmology science
2024

Wide-field optical coherence tomography-angiography in familial exudative vitreoretinopathy.

Journal francais d'ophtalmologie
2024

Familial exudative vitreoretinopathy (FEVR) in a child with a Jagged 1 variant identified on genetic testing.

Ophthalmic genetics
2024

Fluorescein Angiography of Floating Retinal Veins.

JAMA ophthalmology
2024

Coats-Like Presentation of Familial Exudative Vitreoretinopathy Associated With a Novel LRP5 Variant.

Ophthalmic surgery, lasers &amp; imaging retina
2024

Unveiling novel LRP5 pathogenic variant in familial exudative vitreoretinopathy: Diverse phenotypic expressions in a mother-daughter duo.

European journal of ophthalmology
2024

Diagnostic Role of Oral Fluorescein Angiography in Pediatric Ambulatory Clinics.

Ophthalmology. Retina
2025

Clinical and genetic characteristics and natural history of Finnish families with familial exudative vitreoretinopathy due to pathogenic FZD4 variants.

Acta ophthalmologica
2024

Multimodal imaging of white preretinal lesions in atypical familial exudative vitreoretinopathy: Case report and literature review.

American journal of ophthalmology case reports
2024

Identification of Novel FZD4 Mutations in Familial Exudative Vitreoretinopathy and Investigating the Pathogenic Mechanisms of FZD4 Mutations.

Investigative ophthalmology &amp; visual science
2024

Investigating the Impact of Dimer Interface Mutations on Norrin's Secretion and Norrin/β-Catenin Pathway Activation.

Investigative ophthalmology &amp; visual science
2024

Characterization of a novel heterozygous frameshift variant in NDP gene that causes familial exudative vitreoretinopathy in female patients.

Molecular genetics and genomics : MGG
2024

Mutations in TSPAN12 gene causing familial exudative vitreoretinopathy.

Human genomics
2024

Retinopathy With Variant of Unknown Significance and Atypical Chorioretinal Coloboma in the Setting of Prematurity.

Ophthalmic surgery, lasers &amp; imaging retina
2024

Deciphering a crucial dimeric interface governing Norrin dimerization and the pathogenesis of familial exudative vitreoretinopathy.

FASEB journal : official publication of the Federation of American Societies for Experimental Biology
2024

Severe isolated exudative vitreoretinopathy caused by biallelic FZD4 variants.

Clinical genetics
2024

Successful Management of Familial Exudative Vitreoretinopathy with a Large Macular Hole Using Inverted Internal Limiting Membrane Flap Technique.

Case reports in ophthalmology
2024

Genetic Characteristics and Clinical Manifestations of Foveal Hypoplasia in Familial Exudative Vitreoretinopathy.

American journal of ophthalmology
2024

Clinical phenotypic spectrum of CTNNB1 neurodevelopmental disorder.

Clinical genetics
2024

Intra-Anterior Chamber Injection of Ranibizumab in Advanced Pediatric Vitreoretinal Diseases.

JAMA ophthalmology
2024

Wound healing and postoperative management in paediatric patients following 27-Gauge Transconjunctival Sutureless Vitrectomy for vitreoretinal conditions.

International wound journal
2023

Pediatric retinal vascular disorders: From translational sciences to clinical practice.

Saudi journal of ophthalmology : official journal of the Saudi Ophthalmological Society
2023

A Literary Pediatric Retina Fellowship With Michael T. Trese, MD.

Ophthalmic surgery, lasers &amp; imaging retina
2023

De novel heterozygous copy number deletion on 7q31.31-7q31.32 involving TSPAN12 gene with familial exudative vitreoretinopathy in a Chinese family.

International journal of ophthalmology
2024

Frameshift variants in the C-terminal of CTNNB1 cause familial exudative vitreoretinopathy by AXIN1-mediated ubiquitin-proteasome degradation condensation.

International journal of biological macromolecules
2023

Genetic detection of two novel LRP5 pathogenic variants in patients with familial exudative vitreoretinopathy.

BMC ophthalmology
2023

Familial Exudative Vitreoretinopathy Initially Diagnosed as Incontinentia Pigmenti in an Asymptomatic Teenager: A Case Report.

Case reports in ophthalmology
2024

EARLY-ONSET OF FAMILIAL EXUDATIVE VITREORETINOPATHY: Clinical Characteristics, Management, and Outcomes.

Retina (Philadelphia, Pa.)
2023

Patients With Dragged Optic Disc Vessels and Retinal Folds: Clinical Features, Multimodal Imaging, and Histopathology.

Ophthalmic surgery, lasers &amp; imaging retina
2024

Whole genome sequencing for inherited retinal diseases in the Korean National Project of Bio Big Data.

Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie
2023

Mechanisms Underlying Rare Inherited Pediatric Retinal Vascular Diseases: FEVR, Norrie Disease, Persistent Fetal Vascular Syndrome.

Cells
2023

[The role of endothelin-1 in the pathogenesis of familial exudative vitreoretinopathy].

Vestnik oftalmologii
2023

Familial Exudative Vitreoretinopathy-Like Phenotype in a Patient With Microcephaly and TUBGCP6 Mutations.

Journal of vitreoretinal diseases
2025

FAMILIAL EXUDATIVE VITREORETINOPATHY WITH NASAL RETINAL INVOLVEMENT: A RARE PRESENTATION.

Retinal cases &amp; brief reports
2023

AN EIGHT-YEAR RETROSPECTIVE STUDY OF THE ETIOLOGIES, CLINICAL CHARACTERISTICS, AND VISUAL OUTCOMES OF PEDIATRIC LAMELLAR MACULAR HOLE.

Retina (Philadelphia, Pa.)
2023

Refractive Status and Biometric Characteristics of Children With Familial Exudative Vitreoretinopathy.

Investigative ophthalmology &amp; visual science
2023

Vessels characteristics in familial exudative vitreoretinopathy and retinopathy of prematurity based on deep convolutional neural networks.

Frontiers in pediatrics
2023

Genetic and clinical characteristics of ZNF408-related familial exudative vitreoretinopathy.

The Journal of international medical research
2023

Familial exudative vitreoretinopathy complicated with macular hole retinal detachment.

Oman journal of ophthalmology
2023

Peripheral and central retinal vascular changes in asymptomatic family members of patients with familial exudative vitreoretinopathy.

Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie
2023

Defective EMC1 drives abnormal retinal angiogenesis via Wnt/β-catenin signaling and may be associated with the pathogenesis of familial exudative vitreoretinopathy.

Genes &amp; diseases
2023

Pattern of choroidal thickness in early-onset high myopia.

Frontiers in medicine
2023

Two types of childhood glaucoma secondary to familial exudative vitreoretinopathy.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2023

Five novel dysfunctional variants in the TSPAN12 gene in familial exudative vitreoretinopathy.

Experimental eye research
2023

Rapid Improvement in Lipid Maculopathy Following Faricimab Therapy in Recalcitrant Familial Exudative Vitreoretinopathy.

Ophthalmic surgery, lasers &amp; imaging retina
2023

CTNNB1-related neurodevelopmental disorder mimics cerebral palsy: case report.

Frontiers in pediatrics
2023

LONG-TERM CLINICAL OUTCOMES AND GENOTYPE-PHENOTYPE CORRELATION IN FAMILIAL EXUDATIVE VITREORETINOPATHY IN A TERTIARY REFERRAL CENTER.

Retina (Philadelphia, Pa.)
2023

The Characteristic of Optical Coherence Tomography Angiography and Retinal Arteries Angle in Familial Exudative Vitreoretinopathy with Inner Retinal Layer Persistence.

Current eye research
2023

Identification of Five Novel Variants in the TSPAN12 Gene in Chinese Families With Familial Exudative Vitreoretinopathy.

Translational vision science &amp; technology
2023

Optical Coherence Tomography and Optical Coherence Tomography Angiography in Pediatric Retinal Diseases.

Diagnostics (Basel, Switzerland)
2023

Loss of Tbx3 in Mouse Eye Causes Retinal Angiogenesis Defects Reminiscent of Human Disease.

Investigative ophthalmology &amp; visual science
2022

Whole exome sequencing revealed novel pathogenic variants in Vietnamese patients with FEVR.

Molecular vision
2023

An SNX31 variant underlies dominant familial exudative vitreoretinopathy-like pathogenesis.

JCI insight
2023

18p Deletion Syndrome With Concurrent Frizzled-4 Mutation: Surgical Management of Bilateral Stage 5 Traction Retinal Detachment.

Ophthalmic surgery, lasers &amp; imaging retina
2023

Vascular features around the optic disc in familial exudative vitreoretinopathy: findings and their relationship to disease severity.

BMC ophthalmology
2022

Optical Coherence Tomography Angiography in Pediatric Retinal Disorders.

Journal of vitreoretinal diseases
2023

Osteoporosis, Fractures, and Blindness Due to a Missense Mutation in the LRP5 Receptor.

Orthopedic research and reviews
2023

Molecular and Cellular Regulations in the Development of the Choroidal Circulation System.

International journal of molecular sciences
2023

Novel Exon 7 Deletions in TSPAN12 in a Three-Generation FEVR Family: A Case Report and Literature Review.

Genes
2024

Asymmetric familial exudative vitreoretinopathy in a premature infant misdiagnosed as retinopathy of prematurity.

Clinical &amp; experimental optometry
2023

Quantitative Analysis of Vascular Abnormalities in Full-Term Infants With Mild Familial Exudative Vitreoretinopathy.

Translational vision science &amp; technology
2023

Avascular Peripheral Retina in Infants.

Turkish journal of ophthalmology
2023

ULTRA-WIDEFIELD OPTICAL COHERENCE TOMOGRAPHY ANGIOGRAPHY IN MILD FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retina (Philadelphia, Pa.)
2023

Familial Exudative Vitreoretinopathy and Systemic Abnormalities in Patients With CTNNB1 Mutations.

Investigative ophthalmology &amp; visual science
2023

Single-cell transcriptomics-based multidisease analysis revealing the molecular dynamics of retinal neurovascular units under inflammatory and hypoxic conditions.

Experimental neurology
2023

Xp11.3 microdeletion causing Norrie disease and X-linked Kabuki syndrome.

American journal of ophthalmology case reports
2023

Phenotype-Based Genetic Analysis Reveals Missing Heritability of KIF11-Related Retinopathy: Clinical and Genetic Findings.

Genes
2023

The Diagnostic Yield of Next Generation Sequencing in Inherited Retinal Diseases: A Systematic Review and Meta-analysis.

American journal of ophthalmology
2023

Abnormalities of the contralateral eye in unilateral congenital anophthalmic or blind microphthalmic patients.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2023

Biometric Variations in High Myopia Associated with Different Underlying Ocular and Genetic Conditions.

Ophthalmology science
2023

A comprehensive functional analysis on the pathogenesis of novel TSPAN12 and NDP variants in familial exudative vitreoretinopathy.

Clinical genetics
2023

Familial exudative vitreoretinopathy (FEVR) in a child with novel microarray-defined deletion of 11q14 previously diagnosed as retinopathy of prematurity (ROP).

Ophthalmic genetics
2023

Dual phenotype: co-occurring Leber congenital amaurosis and familial exudative vitreoretinopathy: a case report.

Ophthalmic genetics
2022

Clinical characteristics and mutation spectrum in 33 Chinese families with familial exudative vitreoretinopathy.

Annals of medicine
2022

Multimodal Retinal Imaging Findings in Two Cousins With VCAN-Related Vitreoretinopathy or Wagner Disease.

Ophthalmic surgery, lasers &amp; imaging retina
2022

Signal Peptide Variants in Inherited Retinal Diseases: A Multi-Institutional Case Series.

International journal of molecular sciences
2022

Decrease of FZD4 exon 1 methylation in probands from FZD4-associated FEVR family of phenotypic heterogeneity.

Frontiers in medicine
2023

A novel splicing variant of VCAN identified in a Chinese family initially diagnosed with familial exudative vitreoretinopathy.

Molecular genetics &amp; genomic medicine
2022

A boy with amblyopia and familial exudative vitreoretinopathy harboring a new mutation of LRP5 and OPA1: A case report.

Frontiers in genetics
2022

Genetic and clinical characteristics of 24 mainland Chinese patients with CTNNB1 loss-of-function variants.

Molecular genetics &amp; genomic medicine
2022

Genomic and phenotypic characterization of 404 individuals with neurodevelopmental disorders caused by CTNNB1 variants.

Genetics in medicine : official journal of the American College of Medical Genetics
2022

LRP5 BIALLELIC MUTATIONS CAUSE A HIGHER INCIDENCE OF SEVERE PHENOTYPE COMPARED WITH LRP5 MONOALLELIC MUTATION.

Retina (Philadelphia, Pa.)
2022

Atypical Adams-Oliver syndrome with typical ocular signs of familial exudative vitreoretinopathy.

International journal of ophthalmology
2024

RETINAL MANIFESTATIONS OF WALKER-WARBURG SYNDROME IN TWO SIBLINGS WITH RXYLT1 MUTATIONS.

Retinal cases &amp; brief reports
2023

Familial exudative vitreoretinopathy with total retinal detachment: Treatment for scleral buckling.

Asian journal of surgery
2022

Severe Familial Exudative Vitreoretinopathy, Congenital Hearing Loss, and Developmental Delay in a Child With Biallelic Variants in FZD4.

JAMA ophthalmology
2022

Long-term clinical prognosis of 335 infant single-gene positive FEVR cases.

BMC ophthalmology
2022

Noninvasive fetal genotyping of single nucleotide variants and linkage analysis for prenatal diagnosis of monogenic disorders.

Human genomics
2022

Novel CTNNB1 variant leading to neurodevelopmental disorder with spastic diplegia and visual defects plus peripheral neuropathy: A case report.

American journal of medical genetics. Part A
2022

Mutation spectrum in a cohort with familial exudative vitreoretinopathy.

Molecular genetics &amp; genomic medicine
2023

Combined X-linked familial exudative vitreoretinopathy and retinopathy of prematurity phenotype in an infant with mosaic turner syndrome with ring X chromosome.

Ophthalmic genetics
2022

Genotype-phenotype associations in familial exudative vitreoretinopathy: A systematic review and meta-analysis on more than 3200 individuals.

PloS one
2022

Commentary: Familial exudative vitreoretinopathy-The masquerade in pediatric retinal disorders.

Indian journal of ophthalmology
2022

Management and surgical outcomes of pediatric retinal detachment associated with familial exudative vitreoretinopathy - Our experience at a tertiary care ophthalmic center in North India.

Indian journal of ophthalmology
2022

Hedgehog Signal Defect Leading to Familial Exudative Vitreoretinopathy-Like Disease and Gastrointestinal Malformation.

Turkish journal of ophthalmology
2022

CTNND1 variants cause familial exudative vitreoretinopathy through the Wnt/cadherin axis.

JCI insight
2022

Ocular manifestations of Chinese patients with copy number variants in the NDP gene.

Molecular vision
2022

Spectrum of Mutations in NDP Resulting in Ocular Disease; a Systematic Review.

Frontiers in genetics
2022

Familial exudative vitreoretinopathy in a 4 generations family of South-East Asian Descendent with FZD4 mutation (c.1501_1502del).

International journal of retina and vitreous
2022

Dry-Lensectomy Assisted Lensectomy in the Management for End-Stage Familial Exudative Vitreoretinopathy Complicated With Anterior Segment Abnormalities.

Frontiers in medicine
2023

Pseudoretinoblastoma: Distribution based on gender, age, and laterality.

European journal of ophthalmology
2022

Foveal hypoplasia and characteristics of optical components in patients with familial exudative vitreoretinopathy and retinopathy of prematurity.

Scientific reports
2023

Changing trends in pseudoretinoblastoma diagnoses: A 10 year review from the United Kingdom.

European journal of ophthalmology
2022

Update on the Phenotypic and Genotypic Spectrum of KIF11-Related Retinopathy.

Genes
2023

Wide-field Fundus Imaging and Fluorescein Angiography Findings in Various Pseudoretinoblastoma Conditions.

Journal of pediatric ophthalmology and strabismus
2022

Planned Preterm Delivery and Treatment of Severe Infantile FEVR With Osteoporosis-Pseudoglioma Syndrome.

Ophthalmic surgery, lasers &amp; imaging retina
2022

A novel frameshift variant in the TSPAN12 gene causes autosomal dominant FEVR.

Molecular genetics &amp; genomic medicine
2022

Coats plus in prematurity.

Ophthalmic genetics
2022

FZD4 in a Large Chinese Population With Familial Exudative Vitreoretinopathy: Molecular Characteristics and Clinical Manifestations.

Investigative ophthalmology &amp; visual science
2023

OCULAR FEATURES ASSOCIATED WITH MUTATIONS IN ATOH7 GENE OVERLAP THOSE WITH FAMILIAL EXUDATIVE VITREORETINOPATHY.

Retinal cases &amp; brief reports
2022

Severe retinal complications in Knobloch Syndrome - Three siblings without clinically apparent occipital defects and a review of the literature.

Ophthalmic genetics
2023

Novel truncating variants in CTNNB1 cause familial exudative vitreoretinopathy.

Journal of medical genetics
2023

NDP-related retinopathies: clinical phenotype of female carriers.

The British journal of ophthalmology
2022

Whole exome sequencing revealed 14 variants in NDP, FZD4, LRP5, and TSPAN12 genes for 20 families with familial exudative vitreoretinopathy.

BMC medical genomics
2022

Identification of Two Novel Variants in the LRP5 Gene that Cause Familial Exudative Vitreoretinopathy.

Genetic testing and molecular biomarkers
2022

Ocular findings associated with FADD deficiency resemble familial exudative vitreoretinopathy.

American journal of ophthalmology case reports
2022

Unfolding falciform folds and their consequences - To touch or not to touch.

Indian journal of ophthalmology
2022

Structure and function of the retina of low-density lipoprotein receptor-related protein 5 (Lrp5)-deficient rats.

Experimental eye research
Ver todos os 405 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

Ainda não temos associações cadastradas para Vitreorretinopatia exsudativa familiar.

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 Vitreorretinopatia exsudativa familiar

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 login

Doenças 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.

  1. KIF11 prevents retinal endothelial ferroptosis in familial exudative vitreoretinopathy by inhibiting phosphorylation-driven PRDX1 phase separation.
    Nature communications· 2026· PMID 41872221mais citado
  2. An induced pluripotent stem cell line (SJTUXHi003-A) derived from a patient with copy number variation in the gene LRP5 causing familial exudative vitreoretinopathy.
    Stem cell research· 2026· PMID 41496279mais citado
  3. Novel nonsense variant of KIF11 in a patient with MCLMR.
    Human genome variation· 2026· PMID 41771856mais citado
  4. A novel variant p.Y250C of FZD4 influences Norrine/&#x3b2;-catenin signaling pathway that associates with familial exudative vitreoretinopathy (FEVR).
    Scientific reports· 2026· PMID 41526591mais citado
  5. Comparative analysis of activation of macrophages/microglia in diabetic retinopathy and Familial Exudative Vitreoretinopathy.
    Biochemistry and biophysics reports· 2026· PMID 41476775mais citado
  6. Novel variant c.428T>C in FZD4 gene in a pedigree affected by familial exudative vitreoretinopathy: clinical, functional, and structural characterization.
    Ophthalmic Genet· 2026· PMID 41981389recente
  7. Surgical outcomes of familial exudative vitreoretinopathy-associated retinal detachment: a systematic review and meta-analysis.
    Int J Retina Vitreous· 2026· PMID 41965841recente
  8. Predicting mild familial exudative vitreoretinopathy with autosomal dominant inheritance using deep learning.
    Sci Rep· 2026· PMID 41957171recente
  9. Complex Retinal Detachment in an Adult Patient With Familial Exudative Vitreoretinopathy: Challenges in Diagnosis and Management.
    J Vitreoretin Dis· 2026· PMID 41943721recente
  10. Outcomes of vitreoretinal surgery for familial exudative vitreoretinopathy: a systematic review and meta-analysis of the current literature.
    Int Ophthalmol· 2026· PMID 41886127recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:891(Orphanet)
  2. MONDO:0019516(MONDO)
  3. GARD:1613(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q5432936(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

Vitreorretinopatia exsudativa familiar
Compêndio · Raras BR

Vitreorretinopatia exsudativa familiar

ORPHA:891 · MONDO:0019516
Prevalência
Unknown
Herança
Autosomal dominant, Autosomal recessive, X-linked recessive
CID-10
H35.0 · Retinopatias de fundo e alterações vasculares da retina
CID-11
Ensaios
1 ativos
Início
Infancy, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0004608
EuropePMC
Wikidata
Papers 10a
DiscussaoAtiva

Nenhuma novidade ainda. O agente esta monitorando.

0membros
0novidades