Search results for "Kabuki"

showing 9 items of 9 documents

Molecular, clinical and neuropsychological study in 31 patients with Kabuki syndrome and KMT2D mutations

2017

IF 3.326; International audience; Kabuki syndrome (KS-OMIM 147920) is a rare developmental disease characterized by the association of multiple congenital anomalies and intellectual disability. This study aimed to investigate intellectual performance in children with KS and link the performance to several clinical features and molecular data. We recruited 31 children with KMT2D mutations who were 6 to 16 years old. They all completed the Weschler Intelligence Scale for Children, fourth edition. We calculated all indexes: the Full Scale Intellectual Quotient (FSIQ), Verbal Comprehension Index (VCI), Perceptive Reasoning Index (PRI), Processing Speed Index (PSI), and Working Memory Index (WMI…

0301 basic medicineMaleAdolescentVisual impairmentDNA Mutational AnalysisIntelligenceneuropsychologyDisease[SDV.GEN] Life Sciences [q-bio]/GeneticsNeuropsychological Testsgenotype-phenotype correlation03 medical and health sciencesIntensive careIntellectual disabilityGene OrderGeneticsmedicineHumansAbnormalities MultipleChildKMT2D mutationGenetics (clinical)AllelesGenetic Association Studies[SDV.GEN]Life Sciences [q-bio]/GeneticsKabuki syndromebusiness.industryWorking memoryNeuropsychologyWechsler Adult Intelligence Scalemedicine.diseaseHematologic Diseases3. Good healthNeoplasm ProteinsDNA-Binding Proteins030104 developmental biologyPhenotypeVestibular DiseasesGenetic LociFaceMutationFemalemedicine.symptombusinessKabuki syndromeClinical psychology
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Kabuki make-up (Niikawa-Kuroki) syndrome: Clinical and radiological observations in two sicilian children

1991

The Authors describe two patients aged 5 and 8, a female and a male, affected by a condition of polymalformations known as Kabuki make-up or Niikawa-Kuroki syndrome, having a neonatal incidence of 1:32,000 in Japan. There are two hypothesis about the apparent rarity of the syndrome in the rest of the world, including the Asian Continent: the first is that it exists, but is infrequently recognized outside Japan and the second is that it is really more frequent in those parts of the world, where ethnic exchanges are uncommon, as it happens in Japan.

MalePediatricsmedicine.medical_specialtyKabukiEthnic groupNiikawa-Kuroki SyndromeBone and BonesIntellectual DisabilitymedicineHumansAbnormalities MultipleRadiology Nuclear Medicine and imagingChildGrowth Disordersbusiness.industryIncidence (epidemiology)Syndromelanguage.human_languageRadiographyItalyEl NiñoChild PreschoolFaceRadiological weaponPediatrics Perinatology and Child HealthlanguageFemaleCongenital diseasebusinessSicilianPediatric Radiology
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Cholesteatoma associated with Kabuki syndrome

2007

Kabuki syndrome (KS), also identified as Kabuki make-up syndrome or Niikawa-Kuroki syndrome, is a multiple malformation/mental retardation syndrome that was described initially in Japan but is now known to occur in many other ethnic groups. The syndrome is characterized by varying degrees of mental retardation, postnatal growth retardation, distinct facial characteristics resembling the Kabuki actor's make-up, cleft or high-arched palate, brachydactyly, scoliosis, and persistence of finger pads. There is no definitive laboratory diagnostic test for KS, thus diagnosis is based on phenotypic presentation and the exclusion of other known syndromes. Familial cases have been reported and autosom…

Settore MED/31 - OtorinolaringoiatriaCholesteatoma Kabuki Middle ear
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Autism spectrum disorder in Kabuki syndrome: clinical, diagnostic and rehabilitative aspects assessed through the presentation of three cases

2015

Kabuki syndrome (KS) (Kabuki make-up syndrome, Niikawa-Kuroki syndrome) is a rare genetic disorder first diagnosed in 1981. Kabuki make-up syndrome (KMS) is a multiple malformation/intellectual disability syndrome that was first described in Japan but is now reported in many other ethnic groups. KMS is characterized by multiple congenital abnormalities: craniofacial, skeletal, and dermatoglyphic abnormalities; intellectual disability; and short stature. Other findings may include: congenital heart defects, genitourinary anomalies, cleft lip and/or palate, gastrointestinal anomalies including anal atresia, ptosis and strabismus, and widely spaced teeth and hypodontia. The KS is associated wi…

Child development disorders pervasiveIntellectual disabilityKabuki Syndrome; Child development disorders pervasive; Intellectual disabilitySettore MED/39 - Neuropsichiatria InfantileKabuki Syndrome
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DNA methylation episignature testing improves molecular diagnosis of Mendelian chromatinopathies

2021

Abstract Purpose Chromatinopathies include more than 50 disorders caused by disease-causing variants of various components of chromatin structure and function. Many of these disorders exhibit unique genome-wide DNA methylation profiles, known as episignatures. In this study, the methylation profile of a large cohort of individuals with chromatinopathies was analyzed for episignature detection. Methods DNA methylation data was generated on extracted blood samples from 129 affected individuals with the Illumina Infinium EPIC arrays and analyzed using an established bioinformatic pipeline. Results The DNA methylation profiles matched and confirmed the sequence findings in both the discovery an…

Chromatinopathies; DNA methylation; EpigeneticsChromatinopathieBiologyEPICDNA sequencingsymbols.namesakemedicineHumansAbnormalities MultipleGenetics (clinical)Sequence (medicine)GeneticsChromatinopathies; DNA methylation; Epigenetics; DNA Methylation; Genome; Humans; Abnormalities Multiple; Hematologic Diseases; Vestibular DiseasesChromatinopathiesGenomeDNA methylationEpigeneticMethylationHematologic Diseasemedicine.diseaseHematologic DiseasesChromatinVestibular DiseasesDNA methylationMendelian inheritancesymbolsEpigeneticsAbnormalitiesKabuki syndromeMultipleHuman
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Orofacial features and medical profile of eight individuals with Kabuki syndrome

2019

Background To evaluate oral, craniofacial and systemic characteristics of eight patients with Kabuki syndrome (KS), aged between 3 and 16 years old. Material and Methods In this retrospective study, medical records of all patients were reviewed for information on family history, growth and development, medications in use, general systemic complications and oral and craniofacial characteristics. Results The medical alterations found included recurrent infections such as pneumonia and otitis media (n = 6), cardiovascular malformations (n = 4), kidney abnormalities (n = 2), epilepsy (n = 2) and visual deficiency (n = 2). The individuals exhibited dental caries (n = 5), agenesis (n = 5), delaye…

Pediatricsmedicine.medical_specialtyAdolescentCleft LipDental CariesAnodontia03 medical and health sciences0302 clinical medicinemedicineMicrodontiaHumansAbnormalities MultipleCraniofacialFamily historyChildGeneral DentistryAnodontiaRetrospective StudiesTooth Abnormalitiesbusiness.industryResearch030206 dentistryEnamel hypoplasia:CIENCIAS MÉDICAS [UNESCO]Medically compromised patients in Dentistrymedicine.diseaseHematologic DiseasesCleft Palatestomatognathic diseasesOtitisVestibular DiseasesOtorhinolaryngologyChild PreschoolFaceAgenesisUNESCO::CIENCIAS MÉDICASSurgerymedicine.symptombusinessKabuki syndromeMedicina Oral Patología Oral y Cirugia Bucal
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Patient with Kabuki syndrome and acute leukemia

2003

Kabuki syndrome is a multiple congenital anomaly/mental retardation syndrome which often involves recurrent infections. There is cumulative evidence of an immunodeficiency in Kabuki patients. We report a 2-year-old girl with typical Kabuki syndrome, who developed acute lymphocytic leukemia. The patient showed low levels of immunoglobulins G and A and a history of recurrent infections, that might indicate an immunodeficiency leading to an increased susceptibility to cancer. The girl was treated according to BFM protocols adapted to the patient's impaired cardiac situation and severe underweight. She achieved continual complete remission. Classical and molecular cytogenetic analyzes did not d…

Pediatricsmedicine.medical_specialtyAcute leukemiabusiness.industryKabukiInfantCancerPrecursor Cell Lymphoblastic Leukemia-Lymphomamedicine.diseaseIntellectual DisabilityImmunopathologyAcute lymphocytic leukemiaImmunologymedicineHumansAbnormalities MultipleFemaleAbnormalitybusinessKabuki syndromeGenetics (clinical)ImmunodeficiencyAmerican Journal of Medical Genetics
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Neonatal hyperinsulinemic hypoglycemia: case report of kabuki syndrome due to a novel KMT2D splicing-site mutation

2020

Abstract Background Persistent neonatal hypoglycemia, owing to the possibility of severe neurodevelopmental consequences, is a leading cause of neonatal care admission. Hyperinsulinemic hypoglycemia is often resistant to dextrose infusion and needs rapid diagnosis and treatment. Several congenital conditions, from single gene defects to genetic syndromes should be considered in the diagnostic approach. Kabuki syndrome type 1 (MIM# 147920) and Kabuki syndrome type 2 (MIM# 300867), can be associated with neonatal hyperinsulinemic hypoglycemia. Patient presentation We report a female Italian (Sicilian) child, born preterm at 35 weeks gestation, with persistent hypoglycemia. Peculiar facial dys…

0301 basic medicineHeterozygotePediatricsmedicine.medical_specialtyFacial dysmorphismNeonatal hypotoniaCase ReportHypoglycemiamedicine.disease_causeDiagnosis DifferentialNervous system malformation03 medical and health sciences0302 clinical medicineHyperinsulinismmedicineHumansAbnormalities MultipleHyperinsulinemic hypoglycemiaPathologicalbusiness.industryNeonatal hypoglycemiaInfant Newbornlcsh:RJ1-570lcsh:Pediatricsmedicine.diseaseHematologic DiseasesNeoplasm ProteinsDNA-Binding ProteinsPhenotype030104 developmental biologyNeonatal hypotoniaItalyVestibular DiseasesFaceMutationGestationFemalebusinessHyperinsulinismKabuki syndromeInfant PrematureNeonatal hypoglycemia030217 neurology & neurosurgeryItalian Journal of Pediatrics
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Clinical and molecular spectrum of renal malformations in Kabuki syndrome.

2013

International audience; OBJECTIVE: To determine the frequency and types of renal malformations, and to evaluate renal function in a cohort of patients with Kabuki syndrome (KS). STUDY DESIGN: Renal ultrasound scans and plasma creatinine measurements were collected from a French cohort of 94 patients with genotyped KS. Renal function was evaluated based on the estimated glomerular filtration rate. A genotype-phenotype study was conducted for renal and urinary tract malformations. RESULTS: Renal malformations were present in 22% of cases, and urinary tract anomalies were present in 15%. Renal malformations were observed in 28% of the MLL2 mutation-positive group and in 0% of the MLL2 mutation…

MalePathologyGenotyping Techniquesurologic and male genital diseasesKidneyCohort Studieschemistry.chemical_compoundChildUltrasonographyHistone Demethylases0303 health sciencesKidney030305 genetics & heredityNuclear ProteinsHypoplasia3. Good healthNeoplasm ProteinsDNA-Binding Proteinsmedicine.anatomical_structureVestibular DiseasesChild PreschoolCreatinineBiological MarkersFemaleFranceAbnormalitiesMultipleCohort studyGlomerular Filtration RateAdultGenetic Markersmedicine.medical_specialtyAdolescentUrinary systemUrologyRenal function03 medical and health sciencesYoung AdultmedicineHumansAbnormalities MultiplePreschoolGenetic Association Studies030304 developmental biologyRetrospective StudiesCreatinine[SDV.GEN]Life Sciences [q-bio]/Geneticsbusiness.industryInfantRetrospective cohort studymedicine.diseaseHematologic DiseaseschemistryFacePediatrics Perinatology and Child Healthbusiness[ SDV.GEN ] Life Sciences [q-bio]/GeneticsKabuki syndromeBiomarkersThe Journal of pediatrics
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