Raras
Buscar doenças, sintomas, genes...
Síndrome West
ORPHA:3451CID-10 · G40.4CID-11 · 8A62.0DOENÇA RARA

Uma síndrome de epilepsia rara caracterizada pelo início de espasmos epilépticos em bebês entre 2 e 12 meses de idade, e, raramente, até os 24 meses. Os bebês podem não apresentar nenhum histórico médico anterior, ou ter um histórico que mostre a causa por trás da condição. A tríade clássica de espasmos epilépticos, hipsarritmia (um tipo de alteração no exame de eletroencefalograma, que mede a atividade cerebral) e parada ou retrocesso no desenvolvimento é historicamente conhecida como Síndrome de West.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Uma síndrome de epilepsia rara caracterizada pelo início de espasmos epilépticos em bebês entre 2 e 12 meses de idade, e, raramente, até os 24 meses. Os bebês podem não apresentar nenhum histórico médico anterior, ou ter um histórico que mostre a causa por trás da condição. A tríade clássica de espasmos epilépticos, hipsarritmia (um tipo de alteração no exame de eletroencefalograma, que mede a atividade cerebral) e parada ou retrocesso no desenvolvimento é historicamente conhecida como Síndrome de West.

Pesquisas ativas
5 ensaios
42 total registrados no ClinicalTrials.gov
Publicações científicas
1.529 artigos
Último publicado: 2026 Apr 8
Medicamentos
5 registrados
GANAXOLONE, CORTICOTROPIN, VIGABATRIN

Tem tratamento?

5 medicamentos registrados
Ver detalhes, fases e interações →
GANAXOLONECORTICOTROPINVIGABATRINPREDNISOLONECANNABIDIOL
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: G40.4
Você se identifica com essa condição?
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Entender a doença

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Sinais e sintomas

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

Partes do corpo afetadas

🧠
Neurológico
43 sintomas
😀
Face
7 sintomas
🫁
Pulmão
4 sintomas
📏
Crescimento
4 sintomas
💪
Músculos
4 sintomas
🦴
Ossos e articulações
3 sintomas

+ 42 sintomas em outras categorias

Características mais comuns

Atraso global grave do desenvolvimento
Tetraplegia espástica
Deficiência intelectual, grave
Atrofia cerebral
Sinofris
Frieza
116sintomas
Sem dados (116)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 116 características clínicas mais associadas, ordenadas por frequência.

Atraso global grave do desenvolvimentoSevere global developmental delay
Tetraplegia espásticaSpastic tetraplegia
Deficiência intelectual, graveIntellectual disability, severe
Atrofia cerebralCerebral atrophy
SinofrisSynophrys

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico1.529PubMed
Últimos 10 anos200publicações
Pico202558 papers
Linha do tempo
2026Hoje · 2026🧪 1992Primeiro 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

8 genes identificados com associação a esta condição.

ARXHomeobox protein ARXDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor (PubMed:22194193, PubMed:31691806). Binds to specific sequence motif 5'-TAATTA-3' in regulatory elements of target genes, such as histone demethylase KDM5C (PubMed:22194193, PubMed:31691806). Positively modulates transcription of KDM5C (PubMed:31691806). Activates expression of KDM5C synergistically with histone lysine demethylase PHF8 and perhaps in competition with transcription regulator ZNF711; synergy may be related to enrichment of histone H3K4me3 in regulatory element

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Lissencephaly, X-linked 2

A classic type lissencephaly associated with abnormal genitalia. Patients have severe congenital or postnatal microcephaly, lissencephaly, agenesis of the corpus callosum, neonatal-onset intractable epilepsy, poor temperature regulation, chronic diarrhea, and ambiguous or underdeveloped genitalia.

OUTRAS DOENÇAS (9)
X-linked lissencephaly with abnormal genitaliaPartington syndromecorpus callosum agenesis-abnormal genitalia syndromedevelopmental and epileptic encephalopathy, 1
HGNC:18060UniProt:Q96QS3
GUF1Translation factor GUF1, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Promotes mitochondrial protein synthesis. May act as a fidelity factor of the translation reaction, by catalyzing a one-codon backward translocation of tRNAs on improperly translocated ribosomes. Binds to mitochondrial ribosomes in a GTP-dependent manner

LOCALIZAÇÃO

Mitochondrion inner membrane

MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 40

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent. DEE40 inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
34.2 TPM
Fibroblastos
26.7 TPM
Músculo esquelético
18.4 TPM
Skin Sun Exposed Lower leg
17.4 TPM
Nervo tibial
16.9 TPM
OUTRAS DOENÇAS (1)
developmental and epileptic encephalopathy, 40
HGNC:25799UniProt:Q8N442
SIK1Serine/threonine-protein kinase SIK1Disease-causing germline mutation(s) inModerado
FUNÇÃO

Serine/threonine-protein kinase involved in various processes such as cell cycle regulation, gluconeogenesis and lipogenesis regulation, muscle growth and differentiation and tumor suppression. Phosphorylates HDAC4, HDAC5, PPME1, SREBF1, CRTC1/TORC1. Inhibits CREB activity by phosphorylating and inhibiting activity of TORCs, the CREB-specific coactivators, like CRTC2/TORC2 and CRTC3/TORC3 in response to cAMP signaling (PubMed:29211348). Acts as a tumor suppressor and plays a key role in p53/TP53

LOCALIZAÇÃO

CytoplasmNucleus

VIAS BIOLÓGICAS (1)
Phosphorylated BMAL1:CLOCK (ARNTL:CLOCK) activates expression of core clock genes
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 30

A form of epileptic encephalopathy, a heterogeneous group of severe early-onset epilepsies characterized by refractory seizures, neurodevelopmental impairment, and poor prognosis. Development is normal prior to seizure onset, after which cognitive and motor delays become apparent.

EXPRESSÃO TECIDUAL(Tecido-específico)
Skin Not Sun Exposed Suprapubic
14.4 TPM
Skin Sun Exposed Lower leg
10.9 TPM
Ovário
4.6 TPM
Fallopian Tube
4.2 TPM
Tecido adiposo
3.5 TPM
INTERAÇÕES PROTEICAS (5)
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 30early-infantile DEE
HGNC:11142UniProt:P57059
CDKL5Cyclin-dependent kinase-like 5Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Mediates phosphorylation of MECP2 (PubMed:15917271, PubMed:16935860). May regulate ciliogenesis (PubMed:29420175)

LOCALIZAÇÃO

NucleusCytoplasm, cytoskeleton, cilium basal bodyCytoplasm, cytoskeleton, microtubule organizing center, centrosome

OUTRAS DOENÇAS (5)
developmental and epileptic encephalopathy, 2X-linked retinoschisisCDKL5 disorderearly-infantile DEE
HGNC:11411UniProt:O76039
PLCB11-phosphatidylinositol 4,5-bisphosphate phosphodiesterase beta-1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Catalyzes the hydrolysis of 1-phosphatidylinositol 4,5-bisphosphate into diacylglycerol (DAG) and inositol 1,4,5-trisphosphate (IP3) and mediates intracellular signaling downstream of G protein-coupled receptors (PubMed:9188725). Regulates the function of the endothelial barrier

LOCALIZAÇÃO

Nucleus membraneCytoplasm

VIAS BIOLÓGICAS (8)
Synthesis of IP3 and IP4 in the cytosolPLC beta mediated eventsG alpha (q) signalling eventsG beta:gamma signalling through PLC betaCa2+ pathway
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 12

A form of epilepsy characterized by frequent tonic seizures or spasms beginning in infancy with a specific EEG finding of suppression-burst patterns, characterized by high-voltage bursts alternating with almost flat suppression phases. Patients may progress to West syndrome, which is characterized by tonic spasms with clustering, arrest of psychomotor development, and hypsarrhythmia on EEG.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Frontal Cortex BA9
16.7 TPM
Brain Caudate basal ganglia
10.1 TPM
Cólon sigmoide
8.6 TPM
Brain Putamen basal ganglia
8.1 TPM
Córtex cerebral
8.0 TPM
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 12malignant migrating partial seizures of infancy
HGNC:15917UniProt:Q9NQ66
ST3GAL3CMP-N-acetylneuraminate-beta-1,4-galactoside alpha-2,3-sialyltransferaseDisease-causing germline mutation(s) inModerado
FUNÇÃO

Catalyzes the formation of the NeuAc-alpha-2,3-Gal-beta-1,4-GlcNAc-, NeuAc-alpha-2,3-Gal-beta-1,3-GlcNAc- and NeuAc-alpha-2,3-Gal-beta-1,3-GalNAc- sequences found in terminal carbohydrate groups of glycoproteins and glycolipids. The highest activity is toward Gal-beta-1,3-GlcNAc and the lowest toward Gal-beta-1,3-GalNAc

LOCALIZAÇÃO

Golgi apparatus, Golgi stack membraneSecreted

VIAS BIOLÓGICAS (9)
Pre-NOTCH Processing in GolgiMaturation of protein 3aMaturation of protein 3aMaturation of spike proteinSialic acid metabolism
MECANISMO DE DOENÇA

Intellectual developmental disorder, autosomal recessive 12

A disorder characterized by significantly below average general intellectual functioning associated with impairments in adaptive behavior and manifested during the developmental period.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
43.3 TPM
Músculo esquelético
28.4 TPM
Cerebelo
22.7 TPM
Córtex cerebral
22.6 TPM
Nervo tibial
21.6 TPM
INTERAÇÕES PROTEICAS (1)
OUTRAS DOENÇAS (2)
intellectual disability, autosomal recessive 12developmental and epileptic encephalopathy, 15
HGNC:10866UniProt:Q11203
GRIN2BGlutamate receptor ionotropic, NMDA 2BDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Component of N-methyl-D-aspartate (NMDA) receptors (NMDARs) that function as heterotetrameric, ligand-gated cation channels with high calcium permeability and voltage-dependent block by Mg(2+) (PubMed:24272827, PubMed:24863970, PubMed:26875626, PubMed:26919761, PubMed:27839871, PubMed:28095420, PubMed:28126851, PubMed:38538865, PubMed:8768735). Participates in synaptic plasticity for learning and memory formation by contributing to the long-term depression (LTD) of hippocampus membrane currents

LOCALIZAÇÃO

Cell membranePostsynaptic cell membraneCell projection, dendriteLate endosomeLysosomeCytoplasm, cytoskeleton

VIAS BIOLÓGICAS (10)
RAF/MAP kinase cascadeRas activation upon Ca2+ influx through NMDA receptorUnblocking of NMDA receptors, glutamate binding and activationLong-term potentiationNegative regulation of NMDA receptor-mediated neuronal transmission
MECANISMO DE DOENÇA

Intellectual developmental disorder, autosomal dominant 6, with or without seizures

A disorder characterized by significantly below average general intellectual functioning associated with impairments in adaptive behavior and manifested during the developmental period. MRD6 additional features may include seizures, hypotonia, abnormal movements, such as dystonia, and autistic features.

EXPRESSÃO TECIDUAL(Tecido-específico)
Brain Frontal Cortex BA9
6.9 TPM
Brain Nucleus accumbens basal ganglia
5.6 TPM
Córtex cerebral
5.1 TPM
Brain Caudate basal ganglia
4.0 TPM
Brain Anterior cingulate cortex BA24
3.8 TPM
OUTRAS DOENÇAS (2)
developmental and epileptic encephalopathy, 27intellectual disability, autosomal dominant 6
HGNC:4586UniProt:Q13224
SPTAN1Spectrin alpha chain, non-erythrocytic 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Fodrin, which seems to be involved in secretion, interacts with calmodulin in a calcium-dependent manner and is thus candidate for the calcium-dependent movement of the cytoskeleton at the membrane

LOCALIZAÇÃO

Cytoplasm, cytoskeletonCytoplasm, cell cortex

VIAS BIOLÓGICAS (10)
RAF/MAP kinase cascadeNCAM signaling for neurite out-growthCOPI-mediated anterograde transportInteraction between L1 and AnkyrinsCaspase-mediated cleavage of cytoskeletal proteins
MECANISMO DE DOENÇA

Developmental and epileptic encephalopathy 5

A disorder characterized by seizures associated with hypsarrhythmia, profound intellectual disability with lack of visual attention and speech development, as well as spastic quadriplegia.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
345.3 TPM
Cerebelo
315.4 TPM
Nervo tibial
201.2 TPM
Brain Frontal Cortex BA9
191.6 TPM
Córtex cerebral
183.4 TPM
OUTRAS DOENÇAS (4)
neuronopathy, distal hereditary motor, autosomal dominant 11developmental and epileptic encephalopathy, 5developmental delay with or without epilepsyspastic paraplegia 91, autosomal dominant, with or without cerebellar ataxia
HGNC:11273UniProt:Q13813

Medicamentos e terapias

GANAXOLONEPhase 4

Mecanismo: GABA-A receptor; anion channel positive allosteric modulator

CORTICOTROPINPhase 4

Mecanismo: Melanocortin receptor 2 agonist

VIGABATRINPhase 4

Mecanismo: Gamma-amino-N-butyrate transaminase inhibitor

PREDNISOLONEPhase 3

Mecanismo: Glucocorticoid receptor agonist

CANNABIDIOLPhase 3

Mecanismo: Cannabinoid CB1 receptor negative allosteric modulator

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

572 variantes patogênicas registradas no ClinVar.

🧬 ARX: NM_139058.3(ARX):c.969dup (p.Leu324fs) ()
🧬 ARX: GRCh38/hg38 Xp22.33-11.4(chrX:251888-42476276)x2 ()
🧬 ARX: NM_139058.3(ARX):c.1124G>A (p.Trp375Ter) ()
🧬 ARX: NM_139058.3(ARX):c.57del (p.Lys19fs) ()
🧬 ARX: NM_139058.3(ARX):c.1321T>C (p.Phe441Leu) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 41 variantes classificadas pelo ClinVar.

39
2
Patogênica (95.1%)
VUS (4.9%)
VARIANTES MAIS SIGNIFICATIVAS
LOC132090427: GRCh38/hg38 16q23.1(chr16:78064581-78164300)x1 [Pathogenic]
CDKL5: NM_001323289.2(CDKL5):c.404-1G>C [Pathogenic/Likely pathogenic]
LOC126806397: GRCh38/hg38 2q24.3(chr2:165383106-167432622)x1 [Pathogenic]
DNM1: NM_004408.4(DNM1):c.590-2A>G [Pathogenic]
SCN2A: NM_001040142.2(SCN2A):c.707C>G (p.Thr236Ser) [Pathogenic]

Vias biológicas (Reactome)

45 vias biológicas associadas aos genes desta condição.

SLIT2 gene expression is stimulated by ISL1 Primary multipotent pancreatic progenitor cell produces trunk bipotent pancreatic progenitor cell Phosphorylated BMAL1:CLOCK (ARNTL:CLOCK) activates expression of core clock genes Transcriptional Regulation by MECP2 Loss of function of MECP2 in Rett syndrome Regulation of MECP2 expression and activity PLC beta mediated events Synthesis of IP3 and IP4 in the cytosol Acetylcholine regulates insulin secretion Ca2+ pathway G alpha (q) signalling events G beta:gamma signalling through PLC beta Fatty Acids bound to GPR40 (FFAR1) regulate insulin secretion Presynaptic function of Kainate receptors Pre-NOTCH Processing in Golgi Keratan sulfate biosynthesis Defective ST3GAL3 causes MCT12 and EIEE15 Sialic acid metabolism Lewis blood group biosynthesis Maturation of protein 3a Maturation of spike protein Maturation of protein 3a Termination of O-glycan biosynthesis Glycosphingolipid biosynthesis EPHB-mediated forward signaling Unblocking of NMDA receptors, glutamate binding and activation Ras activation upon Ca2+ influx through NMDA receptor RAF/MAP kinase cascade Neurexins and neuroligins Synaptic adhesion-like molecules MECP2 regulates neuronal receptors and channels Activated NTRK2 signals through FYN Assembly and cell surface presentation of NMDA receptors Negative regulation of NMDA receptor-mediated neuronal transmission Long-term potentiation Caspase-mediated cleavage of cytoskeletal proteins Nephrin family interactions NCAM signaling for neurite out-growth Interaction between L1 and Ankyrins Neutrophil degranulation COPI-mediated anterograde transport RHOU GTPase cycle RHOV GTPase cycle Sensory processing of sound by inner hair cells of the cochlea Sensory processing of sound by outer hair cells of the cochlea

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

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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.
Aprovado3
3Fase 35
2Fase 21
·Pré-clínico4
Medicamentos catalogadosEnsaios clínicos· 5 medicamentos · 8 ensaios
✓ Aprovados — podem ser usados hoje
GANAXOLONECORTICOTROPINVIGABATRIN
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Síndrome West

🗺️

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

4 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

42 ensaios clínicos encontrados, 5 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

🥇Melhor nível de evidência: Meta-análise
Timeline de publicações
664 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 664

#1

Developmental and Epileptic Encephalopathy due to Cyclin-Dependent Kinase-Like 5 Deficiency: A Single-Center Experience Across Sex Differences.

Pediatric neurology2026 Apr

The cyclin-dependent kinase-like 5 deficiency disorder (CDD) is an ultrarare X-linked disorder causing early-onset epileptic encephalopathy and severe developmental deficits. Few studies exist on its electroclinical features, outcomes, sex differences, and neuroimaging, particularly from India. This study aims to describe the electroclinical syndrome, developmental profile, radiological findings, and outcomes in patients with CDD and to compare these factors between males and females. This is a hospital-based observational study of patients diagnosed with CDD identified from a prospectively maintained registry of children with developmental and epileptic encephalopathy. Data on demographics, seizure types, epilepsy syndromes, antiseizure medications, electroencephalography findings, developmental assessments, genetic characteristics, brain magnetic resonance imaging, and outcomes were collected. We included 12 patients with pathogenic (9) and likely pathogenic (3) variants in cyclin-dependent kinase-like 5 (CDKL5), among whom seven were female. The mean age at onset of seizures was 5.95 ± 5.56 months and was higher for males than females (8.6 ± 7.23 vs 3.19 ± 2.47). The most common seizure types at onset were tonic seizures in 6 (50%) children and epileptic spasms in 4 (33.3%). Lennox-Gastaut syndrome and West syndrome were the most frequent epilepsy syndromes. The median number of seizures per person was 2.9, and the median number of antiseizure medications used was 6 during their lifetime. Magnetic resonance imaging revealed cerebral volume loss in 7 children and white matter lesions in 6. Severe developmental deficits, a Rett-like phenotype, and cortical visual impairment were observed in three-fourths of the children, and regression of milestones occurred in two-thirds. Repetitive motor behavior (P 0.0455) and regression (P 0.0101) were more common in females. CDD causes refractory epilepsy and severe developmental deficits irrespective of the sex of the patient, variant type, and treatment.

#2

The global research hotspots and future trends of infantile epileptic spasms syndrome: A bibliometric analysis of trends and themes.

Brain & development2026 Feb

This study aims to investigate research trends, key contributors, and emerging topics in the field of infantile epileptic spasms syndrome (IESS). Publications on IESS from 1954 to 2024 were retrieved from the Web of Science Core Collection (WoSCC) database. Bibliometric analysis was performed using Microsoft Excel, VOSviewer, CiteSpace, and R version 4.3.3. A total of 2905 publications were identified, revealing a marked increase in research output from 2000 onward. The USA led with 760 publications. The University of California System was the most productive institution, contributing 420 papers. Epilepsia was the most influential journal, publishing 371 papers with 12,246 citations. Leading authors included Naomichi Matsumoto and Ingrid E. Scheffer. Keywords formed five thematic clusters: (1) genetic foundations and molecular mechanisms (e.g., "mutations"), (2) therapeutic strategies for seizure control (e.g., "ketogenic diet"), (3) epidemiological patterns and seizure classification (e.g., "classification"), (4) clinical practices and treatment outcomes (e.g., "vigabatrin"), and (5) brain structure and diagnostic imaging (e.g., "MRI"). Burst keyword analysis indicated a focus on terms including "encephalopathy", "epileptic spasms", "intellectual disability", "ilae commission", "hypsarrhythmia", "classification", "multicenter", and "management". The findings highlight current hotspots spanning genetic mechanisms, therapeutic strategies, epidemiological patterns, clinical practices, and neuroimaging. Future research should optimize treatments, improve diagnostics, and address developmental impacts.

#3

Cholinergic modulation of hippocampal CA1 pyramidal cell excitability in ArxGCG+7 mice.

Experimental neurology2026 Mar

Developmental and Epileptic Encephalopathies (DEE) are rare neurodevelopmental disorders defined by seizures, developmental delays, and abnormal EEG patterns. Infantile Spasms Syndrome (ISS), the most common DEE, was one of the first linked to single-gene variants, including polyalanine expansions in the Aristaless Related Homeobox (ARX) gene. The ArxGCG+7 mouse, which models the human mutation, exhibits behavioral phenotypes, epileptiform hippocampal activity, and reduced cholinergic input to the hippocampus. The role of the disrupted cholinergic signaling has not been explored in this model and may be contributing to hippocampal dysfunction and DEE pathogenesis. Hence, we investigated the impact of the ARX GCG expansion on the septo-hippocampal network by first reproducing the changes in Choline acetyltransferase (ChAT) protein levels in the medial septum (MS) and diagonal band of Broca (DBB). Next, we performed ex vivo patch-clamp recordings in CA1 pyramidal neurons and measured responses to cholinergic agonists. Finally, we assessed acetylcholine receptor expression by western blot across the hippocampus to assess for homeostatic changes in receptors. Our data demonstrated a ∼ 60 % reduction in ChAT expression in the MS and DBB of ArxGCG+7 mice. The ArxGCG+7 CA1 pyramidal neurons exhibited increased firing activity and reduced sag potential. Despite reduced ChAT, carbachol- and pilocarpine-induced firing responses were preserved, while nicotinic receptor-mediated excitation was abolished. Carbachol-induced depolarization block threshold was elevated in mutants. Molecular analysis revealed increased muscarinic receptor 1 expression and decreased β4 nicotinic receptor expression in CA1.Together, these findings demonstrate histological, electrophysiological, and molecular alterations in the septo-hippocampal network of ArxGCG+7 mice. Together, they suggest impaired cholinergic signaling contributes to ISS pathogenesis and highlight the cholinergic system as a potential therapeutic target.

#4

Treatment Practices for Infantile Epileptic Spasms Syndrome: Consensus and Variation in Major Pediatric Epilepsy Centers.

Pediatric neurology2026 Jan

Infantile epileptic spasms syndrome (IESS) is a developmental and epileptic encephalopathy that requires prompt, effective treatment to optimize outcomes. While the first therapies for IESS with adrenocorticotrophic hormone, prednisolone, or vigabatrin are widely established as a standard, we hypothesized that the treatment protocols of how these therapies should be implemented varied across medical centers. The Pediatric Epilepsy Research Consortium Infantile Spasms Special Interest Group distributed a REDCap survey to 75 US epilepsy centers. Predefined treatment pathway characteristics were extracted and compared. Standard therapy regimens were defined before data collection. Thirty-six centers participated (48% completion rate). Most (89%, n = 32) had IESS treatment pathways, with 72% (n = 23) influenced by insurance barriers such as prior authorizations. Of these, 75% (n = 24) contributed pathways for analysis. Most protocols (88%, n = 21) recommended a standard treatment course for new-onset IESS. Of these, 63% (n = 15) endorsed a sequential approach to using hormonal therapy and vigabatrin, while 17% (n = 4) recommended combination therapy with both for all children. Thirteen centers (54%) provided recommendations for treating persistent epileptic spasms. Approaches to side-effect mitigation varied widely, with gastrointestinal prophylaxis and blood pressure control being the most common (79%, n = 19). Half of the pathways mentioned ketogenic diet (58%) or epilepsy surgery (46%). While there was broad consensus regarding first and second therapy treatment for IESS, variability existed in using sequential versus combination therapy, third therapies, and adverse event monitoring. These findings will guide next research steps in defining key questions on sequential versus combination therapy, third line therapy, and adverse event monitoring in order to develop a standardized consensus-driven treatment protocol for IESS in the future.

#5

Differential hypercapnia: Recognition, management, and outcomes in an awake patient supported with peripheral veno-arterial extracorporeal membrane oxygenation.

JHLT open2026 May

Veno-arterial extracorporeal membrane oxygenation (VA-ECMO) is a life-saving intervention for individuals with refractory cardiopulmonary failure and is associated with several complications. Differential hypercapnia, also known as RIDDLER syndrome or East-West Syndrome, is a complication, at times exacerbated iatrogenically, where competitive flow dynamics lead to relative increase in carbon dioxide delivery from the descending aorta in awake patients supported with peripheral VA-ECMO. This case report presents the clinical course, diagnostic features, management, and outcome for a patient who developed differential hypercapnia during awake peripheral VA-ECMO cannulation and support.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC684 artigos no totalmostrando 197

2026

Differential hypercapnia: Recognition, management, and outcomes in an awake patient supported with peripheral veno-arterial extracorporeal membrane oxygenation.

JHLT open
2026

Clinical and Therapeutic Strategies for West Syndrome in Low-Resource Settings: A 10-Year Experience From Cameroon.

Journal of paediatrics and child health
2026

Clinical characteristics and treatment outcomes of epileptic spasms: Comparative analysis of 277 patients by age at onset (<1 year, 1-2 years, >2 years).

Seizure
2026

Recurrent IRF2BPL c.2152del Variant in NEDAMSS: A Case Report and Comparative Analysis.

American journal of medical genetics. Part A
2026

Case report: A systematic approach for the forensic evaluation of child abuse: lessons from a complex case.

Frontiers in psychiatry
2026

Changes in hypsarrhythmia in West syndrome following combined high-dose prednisolone and vigabatrin therapy: A standardized, low-resolution, brain electromagnetic tomography study.

Medicine
2026

Maternal Child-Directed Speech Toward Children With Infantile Spasm or West Syndrome.

International journal of language &amp; communication disorders
2026

The ketogenic diet alters microbiome-metabolome profiles to improve West syndrome therapy.

Pediatric investigation
2026

Spindle density relates to cognitive outcomes in infantile epileptic spasms syndrome with unknown etiology: A retrospective cohort study.

Epilepsia
2026

Developmental and Epileptic Encephalopathy due to Cyclin-Dependent Kinase-Like 5 Deficiency: A Single-Center Experience Across Sex Differences.

Pediatric neurology
2026

Cannabidiol against Epilepsy: Insights and an Experimental In Silico Approach.

Current pharmaceutical design
2026

'Pseudo-ataxic' negative myoclonic status in developmental and epileptic encephalopathy with spike-wave activation in sleep: Utility of ACTH therapy beyond west syndrome.

Epileptic disorders : international epilepsy journal with videotape
2025

Microphthalmia and Infantile Spasms Leading to the Diagnosis of Aicardi Syndrome: A Case Report and Literature Review of a Rare Entity.

Cureus
2026

Clinical and genotypic characteristics of 19 children with STXBP1-encephalopathy.

Medicine
2026

The global research hotspots and future trends of infantile epileptic spasms syndrome: A bibliometric analysis of trends and themes.

Brain &amp; development
2025

Infantile Spasms (West Syndrome): Integrating Genetic, Neurotrophic, and Hormonal Mechanisms Toward Precision Therapy.

Medicina (Kaunas, Lithuania)
2025

Distributed EEG source localization of hypsarrhythmia in west syndrome: a standardized, low-resolution, brain electromagnetic tomography (sLORETA) study.

BMC neurology
2026

Cholinergic modulation of hippocampal CA1 pyramidal cell excitability in ArxGCG+7 mice.

Experimental neurology
2025

The Enigma of West Syndrome: A Case of Infantile Spasms Without Genetic Clues.

Case reports in pediatrics
2025

[Epileptic encephalopathy associated with a mutation in the KCNT1 gene].

Zhurnal nevrologii i psikhiatrii imeni S.S. Korsakova
2025

Outcomes in West syndrome: Association of genetic and perinatal etiologies with early diagnosis and therapy.

Pediatrics international : official journal of the Japan Pediatric Society
2025

History of Lennox-Gastaut Syndrome: An electro-clinical voyage in search of an epileptic syndrome.

Seizure
2026

Treatment Practices for Infantile Epileptic Spasms Syndrome: Consensus and Variation in Major Pediatric Epilepsy Centers.

Pediatric neurology
2025

Genetic Etiology of Developmental and Epileptic Encephalopathy in a Turkish Cohort: A Single-Center Study with Targeted Gene Panel and Whole Exome Sequencing.

Genes
2025

Genetic testing for infantile-onset epilepsies in resource-limited settings.

Seminars in pediatric neurology
2025

Seizure evolution in a mouse model of West syndrome involves complex and time-dependent synapse remodeling, gliosis and alterations in lipid metabolism.

PLoS biology
2025

A Proof-of-Concept Study on Effectiveness of Nitrazepam in Resistant Infantile Epileptic Spasms Syndrome (NitRIS Trial).

Pediatric neurology
2025

Prenatal betamethasone-postnatal N-methyl-D-aspartic acid model of spasms: Update on mechanisms and treatments.

Epilepsia open
2025

Dose optimization of NMDA for rat model of infantile spasms: Approach using EEG, behavior (Seizure) and histopathology.

Behavioural brain research
2025

Corticotherapy versus adrenocorticotropic hormone for treating West syndrome: a systematic review and meta-analysis.

Arquivos de neuro-psiquiatria
2025

Imaging Findings of Vigabatrin-Associated Neurotoxicity in a 12-Month-Old With Infantile Epileptic Spasm Syndrome.

Cureus
2025

A 60-year follow-up of a case of symptomatic west syndrome transitioned to temporal lobe epilepsy in adulthood.

Epileptic disorders : international epilepsy journal with videotape
2025

Adrenocorticotropic hormone combined with vigabatrin as a second-line therapy for West syndrome.

Arquivos de neuro-psiquiatria
2025

Frontiers in EEG as a tool for the management of pediatric epilepsy: Past, present, and future.

Epilepsia open
2025

Association between phenotypes and genotype of developmental and epileptic encephalopathy in next-generation sequencing methods in infants: A scoping review.

The Medical journal of Malaysia
2025

HECW2 Gene Mutation: A Rare Cause of West Syndrome: A Case Report.

Neurology India
2025

Efficacy and tolerability of magnesium sulfate in children with infantile epileptic spasms syndrome: A systematic review and meta-analysis.

Epilepsy research
2025

Microstructural injury to the optic nerve with vigabatrin treatment in West syndrome: A DTI study.

Scientific reports
2025

Infantile epileptic spasms syndrome as a new phenotype in TOP2B deficiency caused by a de novo variant: a case report and literature review.

Frontiers in pediatrics
2025

Safety, Efficacy, and Tolerability of Ketogenic Diet Versus Adrenocorticotropic Hormone in Infantile Epileptic Spasms Syndrome: A Randomized Controlled Trial.

Pediatric neurology
2025

Quality of life outcomes and predictors for youth with intellectual disability and rare diseases.

Research in developmental disabilities
2025

Endocrinological study of low-dose adrenocorticotropic hormone therapy without tapering in infantile epileptic spasms syndrome.

Brain &amp; development
2025

SCN2A gene mutations with epilepsy: single center experience.

Italian journal of pediatrics
2025

Association Between Scalp High-Frequency Oscillations and Burden of Amplitudes and Epileptiform Discharges (BASED) Scores in Infantile Epileptic Spasms Syndrome.

Biomolecules
2025

Different mutations in TBL1XR1 lead to diverse phenotypes of neurodevelopmental disorder: two case reports.

BMC medical genomics
2025

Connectivity Analysis of Hypsarrhythmia-EEG for Infants With West Syndrome.

IEEE transactions on neural systems and rehabilitation engineering : a publication of the IEEE Engineering in Medicine and Biology Society
2025

A case of early epileptic encephalopathy caused by new mutation at W218C in KCNQ2 and review literature.

Biochemistry and biophysics reports
2025

Pediatric cerebral arteriovenous malformation diagnosed 11 years after neonatal thalamic hemorrhage: illustrative case.

Journal of neurosurgery. Case lessons
2025

Volumetric and microstructural changes in the Hippocampus and cingulum in children with west syndrome.

Brain &amp; development
2025

The Oral Findings and Dental Management of Patients with West Syndrome: A Case Series and Literature Review.

Journal of clinical medicine
2024

Electroclinical characteristics and therapies of tonic spasms.

Acta epileptologica
2025

Infantile Epileptic Spasms Syndrome Due to Neonatal Hypoglycemic Brain Injury: A Retrospective Audit.

Indian pediatrics
2025

COVID-19 infection and vaccination in children with Dravet syndrome or infantile epileptic spasms syndrome: An internet survey in Japan.

Brain &amp; development
2025

Combination Therapy With Vigabatrin and Prednisolone Versus Vigabatrin Alone for Infantile Spasms.

Annals of clinical and translational neurology
2025

Identification of topological alterations using microstate dynamics in patients with infantile epileptic spasms syndrome.

Scientific reports
2025

Relationship between the time course of Burden of Amplitudes and Epileptiform Discharges scores and relapse in children with infantile epileptic spasms syndrome.

Epilepsia
2025

A Novel KMT2E Splicing Variant as a Cause of O'Donnell-Luria-Rodan Syndrome With West Syndrome: Expansion of the Phenotype and Genotype.

International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience
2025

Evidence of thalamocortical network activation during epileptic spasms: A thalamic stereotactic EEG study.

Epilepsia
2025

Disparities in the utilization of genetic testing for non-acquired infantile epileptic spasms syndrome in a single healthcare center in North Carolina.

Epilepsia open
2025

Evaluating the efficacy of very high-dose oral prednisolone in West syndrome: in Uttar Pradesh, India, a resource-limited setting.

Journal of tropical pediatrics
2025

Infantile Epileptic Spasms Syndrome: Unveiling clinical and genetic variability in a case series from Argentina.

Seizure
2024

Diagnostic efficiency of exome-based sequencing in pediatric patients with epilepsy.

Frontiers in genetics
2025

Mild Malformation of Cortical Development With Oligodendroglial Hyperplasia and Epilepsy: A Systematic Review.

Neurology. Genetics
2025

Relatively Increased CO2 Delivered to the Brain From the Descending Aorta Leading to an Elevated Respiratory Rate Causing Differential Hypocapnia (RIDDLER or East-West Syndrome): New Pitfalls in Awake Peripheral V-A ECMO.

Circulation
2025

Williams-Beuren Syndrome and Epilepsy: A Retrospective Analysis of 589 Patients.

Journal of child neurology
2025

Protein-Losing Enteropathy Following Adrenocorticotropic Hormone Therapy in an Infant with West Syndrome.

The Tohoku journal of experimental medicine
2025

Ventricular tachycardia induced by adrenocorticotropic hormone therapy in infantile epileptic spasms syndrome: A case report.

Seizure
2025

Short-Term Effectiveness and Tolerability of Vigabatrin Therapy in Infantile Epileptic Spasms Syndrome.

Indian journal of pediatrics
2025

Severe congenital hyperinsulinism with progressive neurological deterioration due to novel HADH-GHSR digenic mutations: the first case report.

Pediatric endocrinology, diabetes, and metabolism
2024

Association between maternal usage of volatile organic compounds and West syndrome, the Japan Environment and Children's study.

Scientific reports
2025

Infantile epileptic spasms syndrome: When spasms come out of the blue.

Epilepsy &amp; behavior : E&amp;B
2025

Recent advances in CYFIP2-associated neurodevelopmental disorders: From human genetics to molecular mechanisms and mouse models.

Brain &amp; development
2025

Efficacy and tolerability of celastrol and edaravone in the multiple-hit rat model of infantile spasms.

Epilepsy &amp; behavior : E&amp;B
2025

Symptomatic vigabatrin-associated MRI toxicity is associated with simultaneous hormonal therapy among patients with infantile spasms.

Epilepsia open
2024

First-choice hormonal therapies for children with infantile epileptic spasms syndrome in South Asia: A network meta-analysis of randomized controlled trials.

Epilepsia open
2024

Effectiveness of vigabatrin for infantile epileptic spasm syndrome categorized by etiologies.

Seizure
2024

Novel Splice Site Pathogenic Variant in STXBP1 Gene in a Child with Intellectual Disability, Epilepsy, and Autism Spectrum Disorder: A Case Report.

Molecular syndromology
2024

Short-term effectiveness and side effects of ketogenic diet for drug-resistant epilepsy in children with genetic epilepsy syndromes.

Frontiers in neurology
2024

Predictive modeling based on functional connectivity of interictal scalp EEG for infantile epileptic spasms syndrome.

Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology
2024

New evidence supports RYR3 as a candidate gene for developmental and epileptic encephalopathy.

Frontiers in neurology
2024

Role of pediatric dentist in West syndrome rehabilitation: A case report.

Special care in dentistry : official publication of the American Association of Hospital Dentists, the Academy of Dentistry for the Handicapped, and the American Society for Geriatric Dentistry
2024

Global and multi-partition local network analysis of scalp EEG in West syndrome before and after treatment.

Neural networks : the official journal of the International Neural Network Society
2024

Epilepsy surgery for children with epileptic spasms: A systematic review and meta-analysis with focus on predictors and outcomes.

Epilepsia open
2024

Radiographic and Tomographic Study of the Cranial Bones in Children with the Idiopathic Type of West Syndrome.

Pediatric reports
2024

The Utility of Genetic Testing in Infantile Epileptic Spasms Syndrome: A Step-Based Approach in the Next-Generation Sequencing Era.

Pediatric neurology
2024

Interictal EEG features as computational biomarkers of West syndrome.

Frontiers in pediatrics
2024

The Association Between Serum Levels of Glial Biomarkers, Clinical Severity and Electro-encephalography Features in Idiopathic West and Lennox-Gastaut Syndromes.

Noro psikiyatri arsivi
2024

Epilepsy, EEG and chromosomal rearrangements.

Epilepsia open
2024

[Clinical and genetic analysis of a child with West syndrome due to a de novo variant of NEXMIF gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2024

Adrenocortical Function Recovery from Secondary Adrenal Insufficiency After ACTH Therapy in a Patient with West Syndrome: A Case Report and Literature Review.

Yonago acta medica
2024

Early Response, Long-Term Seizure Outcome, and Very-Low-Dose Adrenocorticotrophic Hormone Therapy for Infantile Epileptic Spasms Syndrome With Down Syndrome.

Pediatric neurology
2024

Computational EEG attributes predict response to therapy for epileptic spasms.

Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology
2024

Underlying Disorders in Children With Infection-Related Acute Encephalopathy.

Pediatric neurology
2024

Epileptic spasms relapse is associated with response latency but not conventional attributes of post-treatment EEG.

Epilepsia open
2024

Electrographic screening for infantile epileptic spasms syndrome in a single sleep-wake cycle.

Epilepsia
2024

Genetic Advancements in Infantile Epileptic Spasms Syndrome and Opportunities for Precision Medicine.

Genes
2024

Epileptic spasms in clusters without hypsarrhythmia in infancy and childhood: A single age-dependent type of epilepsy or well-defined epileptic syndrome?

Epilepsy research
2024

Early-onset West syndrome with developmental delay associated with a novel KLHL20 variant.

American journal of medical genetics. Part A
2024

Metabolic etiologies in children with infantile epileptic spasm syndrome: Experience at a tertiary pediatric neurology center.

Brain &amp; development
2024

Electroretinogram as a Screening Tool to Assess Vigabatrin-Induced Retinal Toxicity in Children With Infantile Spasms.

Journal of pediatric ophthalmology and strabismus
2024

A child with TSC2/PKD1 contiguous gene deletion syndrome successfully treated with tolvaptan for rapidly enlarging renal cysts.

CEN case reports
2025

Electroclinical Landscape of Infantile Epileptic Spasms Syndrome.

Indian journal of pediatrics
2024

A Pair of Compound Heterozygous IARS2 Variants Manifesting West Syndrome and Electrolyte Disorders in a Chinese Patient.

Global medical genetics
2024

Metabolic causes of pediatric developmental & epileptic encephalopathies (DEE)- genetic variant analysis in a south Indian cohort.

Seizure
2023

Immunological markers of drug resistant epilepsy and its response to immunomodulatory therapy with ACTH in children.

Folia neuropathologica
2024

Societal costs of illness for infantile epileptic spasms syndrome and evolutionary cost prediction in the era of WHO's IGAP.

Epilepsy research
2024

Common genes and recurrent causative variants in 957 Asian patients with pediatric epilepsy.

Epilepsia
2024

The first report of a Korean/Vietnamese child with novel pathogenic variants in Asparagine Synthetase Deficiency (ASNSD) with evolving epilepsy syndromes.

Seizure
2023

Proteomic analysis of salivary inflammatory biomarkers of developmental gingival enlargements in patients with West and Noonan syndromes: a preliminary pilot single-center retrospective study.

European review for medical and pharmacological sciences
2023

Effective Epilepsy Surgery for Post-Traumatic West Syndrome Following Abusive Head Trauma.

Acta medica Okayama
2024

Fifteen years of real-world data on the use of vigabatrin in individuals with infantile epileptic spasms syndrome.

Epilepsia
2024

Treatment modalities for infantile spasms: current considerations and evolving strategies in clinical practice.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2023

Three-Year Follow-Up after Intrauterine mTOR Inhibitor Administration for Fetus with TSC-Associated Rhabdomyoma.

International journal of molecular sciences
2023

Rhabdomyolysis during ACTH therapy for west syndrome.

Pediatrics international : official journal of the Japan Pediatric Society
2023

Distribution of peripheral blood mononuclear cell subtypes in patients with West syndrome: Impact of synacthen treatment.

Immunology letters
2023

Care of Children with Infantile Epileptic Spasms Syndrome and Applicability of Telemedicine Amidst the COVID-19 Pandemic.

Indian journal of pediatrics
2023

Disruption of mitochondrial and lysosomal functions by human CACNA1C variants expressed in HEK 293 and CHO cells.

Frontiers in molecular neuroscience
2023

Electrophysiological network predicts clinical response to vigabatrin in epileptic spasms.

Frontiers in neurology
2023

The landscape of infantile epileptic spasms syndrome in South Asia: peculiarities, challenges, and way forward.

The Lancet regional health. Southeast Asia
2023

Inaccuracies in Parental Reporting of Treated Epileptic Spasms: Both Under- and Over-Reporting.

Pediatric neurology
2023

Nutritional vitamin B12 deficiency-associated Infantile epileptic spasms syndrome: Clinico-neurophysiological presentation, response to treatment, and neurodevelopmental outcome.

Seizure
2023

Altered serum levels of platelet-derived growth factor receptor β and cluster of differentiation 13 suggest a role for pericytes in West syndrome.

Brain &amp; development
2023

Modified Atkins Diet vs. Ketogenic Diet in the Management of Children with Epileptic Spasms Refractory to First Line Treatment: An Open Labelled, Randomized Controlled Trial.

Indian journal of pediatrics
2023

Clinico-Etiologic Profile of Children and Adolescents with Drug-Resistant Epilepsy in a low-Resource Setting: 10 Years' Experience.

Journal of child neurology
2023

A case of West syndrome and global developmental delay in a child with a heterozygous mutation in the TBL1XR1 gene: A case report.

Medicine
2023

Video-based detection of epileptic spasms in West syndrome using a deep neural network: A pilot case study.

Journal of the neurological sciences
2024

Early motor repertoire and developmental function outcomes in infants with West syndrome: a case series.

Physiotherapy theory and practice
2023

Impact of COVID-19 Infection in West Syndrome: The Need for More Data.

Indian journal of pediatrics
2023

Ohtahara and West Syndrome due to Pyridox(am)ine-5-Phosphate Oxidase (PNPO) Deficiency with Novel Phenotype and Good Outcome without Pyridoxal-5'-Phosphate.

Indian journal of pediatrics
2022

Phenotypic Pleiotropy in Arginase Deficiency: A Single Center Cohort.

Annals of Indian Academy of Neurology
2023

Cost-effectiveness of adrenocorticotropic hormone injection and oral prednisolone in patients with West syndrome: A comparative analysis.

Journal of neurosciences in rural practice
2023

The Electroencephalographic Characterization of Hypsarrhythmia in Older Pediatric Population With Epilepsy Using Computer-Added Quantitative Methods.

Cureus
2022

Evaluation of immunological abnormalities in patients with rare syndromes.

Central-European journal of immunology
2023

The Epilepsy Surgery Experience in Children With Infantile Epileptic Spasms Syndrome at a Tertiary Care Center in Canada.

Journal of child neurology
2023

Paradoxical spells during ACTH treatment in an infant with Tetralogy of Fallot.

Pediatrics international : official journal of the Japan Pediatric Society
2023

Extended Glasgow Outcome Scale to Evaluate the Functional Impairment of Patients With Subcortical Band Heterotopia: A Multicentric Cross-sectional Study.

Pediatric neurology
2022

Persistent hyperplastic primary vitreous in a child with incontinentia pigmenti and infantile spasms.

Oman journal of ophthalmology
2023

Genotype and phenotype characteristics of West syndrome in 20 Vietnamese children: Two novel variants detected by next-generation sequencing.

Epilepsy research
2023

[Current Position of Callosotomy].

No shinkei geka. Neurological surgery
2022

Retrospective Clinical Analysis of Epilepsy Treatment for Children with Drug-Resistant Epilepsy (A Single-Center Experience).

Brain sciences
2023

Biochemical mechanisms in pathogenesis of infantile epileptic spasm syndrome.

Seizure
2023

Prednisolone or tetracosactide depot for infantile epileptic spasms syndrome? A prospective analysis of data embedded within two randomised controlled trials.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
2023

Cataract surgery outcomes in pediatric patients with systemic comorbidities.

Indian journal of ophthalmology
2022

West Syndrome and the Importance of Routine Physical Examinations and Parental Education: A Case Report.

Cureus
2022

Clinical and genetic study of developmental and epileptic encephalopathy in Argentinean pediatric patients.

Medicina
2022

Adjunctive perampanel therapy for patients with epileptic spasms.

Pediatrics international : official journal of the Japan Pediatric Society
2022

Finding the Best: Corticosteroids for the Treatment of West Syndrome.

Annals of Indian Academy of Neurology
2022

Oral Dexamethasone versus Prednisolone for Management of Children with West Syndrome: An Open-Labeled Randomized Controlled Pilot Trial.

Annals of Indian Academy of Neurology
2022

Emerging Verbal Functions in Early Infancy: Lessons from Observational and Computational Approaches on Typical Development and Neurodevelopmental Disorders.

Advances in neurodevelopmental disorders
2022

Infantile epileptic spasms syndrome as an initial presentation in infantile choroid plexus papilloma: A case report.

Frontiers in pediatrics
2022

Genotype and phenotype spectrum of 10 children with STXBP1 gene-related encephalopathy and epilepsy.

Frontiers in pediatrics
2023

Partial Efficacy of Vigabatrin in an Infant With West Syndrome Due to Pyruvate Dehydrogenase Complex Deficiency: A Case Report.

Pediatric neurology
2022

A case of carbonic anhydrase type VA deficiency presenting as West syndrome in an infant with a novel mutation in the CA-VA gene.

Epilepsy &amp; behavior reports
2022

Efficacy of pulse intravenous methylprednisolone in epileptic encephalopathy: a randomised controlled trial.

Journal of neurology, neurosurgery, and psychiatry
2022

Quantitative pretreatment EEG predicts efficacy of ACTH therapy in infantile epileptic spasms syndrome.

Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology
2022

Periodic electroencephalographic discharges and epileptic spasms involve cortico-striatal-thalamic loops on Arterial Spin Labeling Magnetic Resonance Imaging.

Brain communications
2022

Mutation in the STXBP1 Gene Associated with Early Onset West Syndrome: A Case Report and Literature Review.

Pediatric reports
2022

A Retrospective Cohort Study of Combined Therapy in West Syndrome associated with Trisomy 21.

Child neurology open
2023

CYFIP2 p.Arg87Cys Causes Neurological Defects and Degradation of CYFIP2.

Annals of neurology
2022

Rates of rare copy number variants in different circumstances among patients with genetic developmental and epileptic encephalopathy.

Science progress
2022

Cerebral small vessel disease caused by PLOD3 mutation: Expanding the phenotypic spectrum of lysyl hydroxylase-3 deficiency.

Pediatric investigation
2022

Neurobehavioral deficits and a progressive ictogenesis in the tetrodotoxin model of epileptic spasms.

Epilepsia
2023

DYNC1H1-related epilepsy: Genotype-phenotype correlation.

Developmental medicine and child neurology
2022

Involvement of the Thalamus, Hippocampus, and Brainstem in Hypsarrhythmia of West Syndrome: Simultaneous Recordings of Electroencephalography and fMRI Study.

AJNR. American journal of neuroradiology
2022

MED13 mutation: A novel cause of developmental and epileptic encephalopathy with infantile spasms.

Seizure
2022

Resective epilepsy surgery for West syndrome: The Hypsarrhythmic Asymmetric Scoring Scheme is a determining predictor of seizure outcome.

Seizure
2022

Adrenocorticotropic Hormone Induced Status Dystonicus in a Child with West Syndrome.

Indian journal of critical care medicine : peer-reviewed, official publication of Indian Society of Critical Care Medicine
2022

Refractory status epilepticus with fever due to mumps vaccine-induced encephalitis caused secondary encephalopathy mimicking acute encephalopathy with biphasic seizures and late reduced diffusion.

Brain &amp; development
2022

Post-vaccination drug-resistant epileptic spasms associated with homozygous IFNAR2 pathogenic variant: Case report✰.

Seizure
2022

Clinical phenotype and genotype of children with GABAA receptor α1 subunit gene-related epilepsy.

Frontiers in neurology
2022

Molecular Insights into the Role of Pathogenic nsSNPs in GRIN2B Gene Provoking Neurodevelopmental Disorders.

Genes
2022

Challenges in Rare Diseases Diagnostics: Incontinentia Pigmenti with Heterozygous GBA Mutation.

Diagnostics (Basel, Switzerland)
2022

Asymmetric epileptic spasms after corpus callosotomy in children with West syndrome may be a good indicator for unilateral epileptic focus and subsequent resective surgery.

Epilepsia open
2022

West Syndrome and the new classification of epilepsy.

The Lancet. Neurology
2022

Missense variant c.1460 T > C (p.L487P) enhances protein degradation of ER mannosyltransferase ALG9 in two new ALG9-CDG patients presenting with West syndrome and review of the literature.

Molecular genetics and metabolism
2022

Monoamine neurotransmitters in early epileptic encephalopathies: New insights into pathophysiology and therapy.

Developmental medicine and child neurology
2022

The effectiveness and tolerability of clobazam in the pediatric population: Adjunctive therapy and monotherapy in a large-cohort multicenter study.

Epilepsy research
2022

Delineating the epilepsy phenotype of NRROS-related microgliopathy: A case report and literature review.

Seizure
2022

Scalp EEG functional connection and brain network in infants with West syndrome.

Neural networks : the official journal of the International Neural Network Society
2023

COVID-19 in Children with West Syndrome: An Ambispective Study.

Indian journal of pediatrics
2022

Rare Cause of West syndrome secondary to Tubulinopathy due to Congenital Symmetric Circumferential Skin Creases (CSCSC) Kunze Type due to a Novel Variant in MAPRE2 Gene.

Annals of Indian Academy of Neurology
2022

Outcome at age 7 of epilepsy presenting in the first 2 years of life. A population-based study.

Epilepsia
2022

The Clinical Features and Long-Term Follow-Up of Vitamin B6-Responsive Infantile Spasms in a Chinese Cohort.

Frontiers in neurology
2022

Retrospective Echocardiographic Analysis of West Syndrome During Adrenocorticotropic Hormone Therapy.

Frontiers in pediatrics
2022

Rare variant of TBL1XR1 in West syndrome: A case report.

Molecular genetics &amp; genomic medicine
2022

Focal seizures during adrenocorticotropic hormone therapy in a school-aged boy: a case report.

Journal of medical case reports
2022

Perampanel markedly improved clinical seizures in a patient with a Rett-like phenotype and 960-kb deletion on chromosome 9q34.11 including the STXBP1.

Clinical case reports
2022

Long-Term Outcome in West Syndrome: The Facts and the Scope to Improve It Further.

Indian journal of pediatrics
2023

Hypsarrhythmia paroxysm intensities that initiate and render physical and mental retardation irreversible in West syndrome.

The International journal of neuroscience
2022

Discrimination of secondary hypsarrhythmias to Zika virus congenital syndrome and west syndrome based on joint moments and entropy measurements.

Scientific reports
2022

Long-term outcome of developmental and epileptic encephalopathies.

Revue neurologique
2021

[The role of "situated storytelling" in therapeutic patient education (TPE): from information to relationship].

Sante publique (Vandoeuvre-les-Nancy, France)
2022

SCN2A-Related Epilepsy: The Phenotypic Spectrum, Treatment and Prognosis.

Frontiers in molecular neuroscience
2022

Extent of EEG monitoring for detecting epileptic spasms.

Seizure
2022

Deep feature fusion based childhood epilepsy syndrome classification from electroencephalogram.

Neural networks : the official journal of the International Neural Network Society
2022

Historical Overview of Hypsarrhythmia and Its Association to Epileptic Spasms: A Review of the Medical Literature From 1952 to 1982.

Journal of clinical neurophysiology : official publication of the American Electroencephalographic Society
2022

Does Etiology and Hypsarrhythmia Subtype Influence Outcome in West Syndrome? Challenges Encountered from a Referral Center Perspective.

Neurology India
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Doenças relacionadas

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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.

  1. Developmental and Epileptic Encephalopathy due to Cyclin-Dependent Kinase-Like 5 Deficiency: A Single-Center Experience Across Sex Differences.
    Pediatric neurology· 2026· PMID 41619470mais citado
  2. The global research hotspots and future trends of infantile epileptic spasms syndrome: A bibliometric analysis of trends and themes.
    Brain &amp; development· 2026· PMID 41546957mais citado
  3. Cholinergic modulation of hippocampal CA1 pyramidal cell excitability in ArxGCG+7 mice.
    Experimental neurology· 2026· PMID 41390005mais citado
  4. Treatment Practices for Infantile Epileptic Spasms Syndrome: Consensus and Variation in Major Pediatric Epilepsy Centers.
    Pediatric neurology· 2026· PMID 41172580mais citado
  5. Differential hypercapnia: Recognition, management, and outcomes in an awake patient supported with peripheral veno-arterial extracorporeal membrane oxygenation.
    JHLT open· 2026· PMID 41852366mais citado
  6. American Epilepsy Society Clinical Practice Guideline: Infantile Epilepsy.
    Epilepsy Curr· 2026· PMID 41969626recente
  7. Clinical and Therapeutic Strategies for West Syndrome in Low-Resource Settings: A 10-Year Experience From Cameroon.
    J Paediatr Child Health· 2026· PMID 41837584recente
  8. Clinical characteristics and treatment outcomes of epileptic spasms: Comparative analysis of 277 patients by age at onset (2 years).
    Seizure· 2026· PMID 41819010recente
  9. Recurrent IRF2BPL c.2152del Variant in NEDAMSS: A Case Report and Comparative Analysis.
    Am J Med Genet A· 2026· PMID 41813602recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:3451(Orphanet)
  2. MONDO:0018097(MONDO)
  3. GARD:7887(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Artigo Wikipedia(Wikipedia)
  7. Q1041258(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.

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ORPHA:3451 · MONDO:0018097
CID-10
G40.4 · Outras epilepsias e síndromes epilépticas generalizadas
CID-11
Ensaios
5 ativos
Medicamentos
5 registrados
MedGen
UMLS
C0037769
EuropePMC
Wikidata
Wikipedia
Papers 10a
Evidência
🥇 Meta-análise
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