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showing 10 items of 2571 documents

Using the Hemophilia Joint Health Score for assessment of children: Reliability of the Spanish version

2018

Introduction: Numerous measuring instruments for the evaluation of hemophilic arthropathy have been developed. One of the most used systems is the Hemophilia Joint Health Score (HJHS) given its sensitivity to clinical changes appearing in the joints because of recurrent hemarthrosis. Objective: Assessing the interrater reliability, using the Spanish version of the HJHS (version 2.1) in children with hemophilia. Design: Reliability study to assess the interrater reliability of the Spanish version of HJHS. Methods: A sample of 36 children aged 7–13 years diagnosed with hemophilia A or B was used. Two physiotherapists performed physical assessments with the Spanish version of the HJHS. Descrip…

Malecongenital hereditary and neonatal diseases and abnormalities030506 rehabilitationmedicine.medical_specialtyAdolescentInterobserver reliabilityHealth StatusHemophilic arthropathyPhysical Therapy Sports Therapy and RehabilitationHemophilia AHemophilia BHemofilia03 medical and health sciences0302 clinical medicinePredictive Value of Testshemic and lymphatic diseasesHemarthrosisHealth Status IndicatorsHumansMedicineHealth scoreChildJoint (geology)Reliability (statistics)Observer Variationbusiness.industryAge FactorsReproducibility of ResultsSpanish versionHemartrosisCross-Sectional StudiesSpainNiñoArtropatíasJoint damagePhysical therapyJointsLucha contra las enfermedades0305 other medical sciencebusiness030217 neurology & neurosurgeryPhysiotherapy Theory and Practice
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Psychosocial adjustment of children with spina bifida.

1998

It was the aim of the present prospective study to investigate the influence of age, sex, intellectual function, and school type as well as of hydrocephalus, the level of lesion, and of the degree of handicap on the psychosocial adjustment of children with spina bifida. Seventy-five patients with spina bifida, aged 6 to 16 years were assessed concerning their psychosocial adjustment and their intellectual function by use of standardized instruments. The findings were compared with those of nondisabled controls, matched for age and sex. Children with spina bifida showed a tendency to be at an increased risk for psychosocial maladjustment. Influencing factors were age, sex, and the degree of…

Malecongenital hereditary and neonatal diseases and abnormalitiesAdolescentAge and sexDevelopmental psychologyIntellectual function03 medical and health sciences0302 clinical medicine030225 pediatricsGermanySurveys and QuestionnairesmedicineHumansProspective StudiesProspective cohort studyChildSpinal DysraphismSchool typeSpina bifidamedicine.diseasenervous system diseasesHydrocephalusIncreased riskPediatrics Perinatology and Child HealthFemaleNeurology (clinical)Test Anxiety ScalePsychologyPsychosocialSocial Adjustment030217 neurology & neurosurgeryClinical psychologyJournal of child neurology
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Subcortical somatosensory evoked potentials after median nerve and posterior tibial nerve stimulation in high cervical cord compression of achondropl…

2008

Abstract Children with achondroplasia may have high cervical myelopathy from stenosis of the cranio-cervical junction resulting in neurological disability and an increased rate of sudden death. To detect myelopathy we recorded somatosensory evoked potentials after median nerve (MN) and posterior tibial nerve (PTN) stimulation in 77 patients with achondroplasia aged 0.3–17.8 years (mean 2.7 years). In addition to the conventional technique of recording the cortical components and the central conduction time (CCT) we employed non-cephalic and mastoid reference electrodes to record the subcortical waveforms N13b and P13 (MN-SEP) as well as P30 (PTN-SEP), respectively, which are generated near …

Malecongenital hereditary and neonatal diseases and abnormalitiesAdolescentSudden deathSensitivity and SpecificityAchondroplasiaMyelopathyDevelopmental NeuroscienceSpinal cord compressionEvoked Potentials SomatosensoryMedicineHumansTibial nerveChildbusiness.industryInfantGeneral MedicineCervical cord compressionAnatomymedicine.diseaseSpinal cordMagnetic Resonance ImagingElectric StimulationMedian Nervebody regionsmedicine.anatomical_structureSomatosensory evoked potentialChild PreschoolPediatrics Perinatology and Child HealthCervical VertebraeFemaleNeurology (clinical)Tibial NervebusinessMyelomalaciaSpinal Cord CompressionBraindevelopment
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Association between the HFE mutations and longevity: a study in Sardinian population

2003

Hereditary hemochromatosis is an HLA-linked inherited disease characterised by inappropriately high absorption of iron by the gastrointestinal mucosa. The cysteine-to-tyrosine substitution at codon 282 of the HFE encoding gene sequence is responsible for the disease, although other variants, as H63D and S65C, may modify the affinity of the protein for transferrin receptors. We have recently reported that C282Y mutation is significantly increased in very old (>90 years) Sicilian women, suggesting a role in attainment of longevity. In addition, an increase of H63D polymorphism was also observed in these women but the difference was not significant. To validate and extend these results we inve…

Malecongenital hereditary and neonatal diseases and abnormalitiesAgingIronLongevityPopulation geneticsTransferrin receptorBiologyPolymorphism (computer science)medicineHumansPoint MutationAlleleHemochromatosis ProteinHemochromatosisAgedAged 80 and overGeneticsPolymorphism GeneticHistocompatibility Antigens Class IMembrane Proteinsnutritional and metabolic diseasesMiddle Agedmedicine.diseaseItalyHereditary hemochromatosisMutation (genetic algorithm)CentenarianDevelopmental BiologyMechanisms of Ageing and Development
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Cytogenetic study of a spindle-cell rhabdomyosarcoma of the parotid gland.

1999

The cytogenetic analysis of a spindle-cell rhabdomyosarcoma of the parotid gland in a 6-year-old boy is reported. The tumor cells showed an abnormal karyotype with a hypotriploid modal chromosome number and clonal structural rearrangements affecting chromosomes 1, 8, 12, 21, and 22. The tumor karyotype was: 59, XY, -1, -3, -4, -5, -6, +8, +8, +del(8)(q22q24), -9, -10, del(12)(q13), -15, -16, -17, -18, der(21)t(12;21)(p11;p11), -22, der(22)t(1;22)(q12;p11).

Malecongenital hereditary and neonatal diseases and abnormalitiesCancer Researchmedicine.medical_specialtyPathologyTransplantation HeterologousMice NudeBiologyMyosinsDesminMiceRhabdomyosarcomaGeneticsmedicineAnimalsChromosomes HumanHumansVimentinRhabdomyosarcomaSpindle cell rhabdomyosarcomaChildMolecular BiologyChromosome AberrationsMyoglobinCytogeneticsKaryotypeModal Chromosome NumberAnatomymedicine.diseaseImmunohistochemistryActinsParotid glandParotid Neoplasmsmedicine.anatomical_structureKaryotypingImmunohistochemistrySarcomaNeoplasm TransplantationCancer genetics and cytogenetics
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Xq27 FRAXA Locus is a Strong Candidate for Dyslexia: Evidence from a Genome-Wide Scan in French Families

2012

Dyslexia is a frequent neurodevelopmental learning disorder. To date, nine susceptibility loci have been identified, one of them being DYX9, located in Xq27. We performed the first French SNP linkage study followed by candidate gene investigation in dyslexia by studying 12 multiplex families (58 subjects) with at least two children affected, according to categorical restrictive criteria for phenotype definition. Significant results emerged on Xq27.3 within DYX9. The maximum multipoint LOD score reached 3,884 between rs12558359 and rs454992. Within this region, seven candidate genes were investigated for mutations in exonic sequences (CXORF1, CXORF51, SLITRK2, FMR1, FMR2, ASFMR1, FMR1NB), al…

Malecongenital hereditary and neonatal diseases and abnormalitiesCandidate geneGenotypeGenome-wide association studyLocus (genetics)BiologyPolymorphism Single NucleotideGenomeDyslexiaFragile X Mental Retardation ProteinGenes X-LinkedGenotypeGeneticsmedicineHumansSNPGenetic Predisposition to DiseaseChildGenetics (clinical)Ecology Evolution Behavior and SystematicsGeneticsChromosomes Human XDyslexiamedicine.diseaseFMR1Settore MED/39 - Neuropsichiatria InfantilePedigreeGenetic LociFemaleFranceDyslexia Linkage study Multiplex families Fmr1 Dyx 9 loci InLod ScoreGenome-Wide Association StudyBehavior Genetics
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Novel deletion of the E3A ubiquitin protein ligase gene detected by multiplex ligation-dependent probe amplification in a patient with Angelman syndr…

2010

Angelman syndrome (AS) is a severe neurobehavioural disorder caused by failure of expression of the maternal copy of the imprinted domain located on 15q11-q13. There are different mechanisms leading to AS: maternal microdeletion, uniparental disomy, defects in a putative imprinting centre, mutations of the E3 ubiquitin protein ligase (UBE3A) gene. However, some of suspected cases of AS are still scored negative to all the latter mutations. Recently, it has been shown that a proportion of negative cases bear large deletions overlapping one or more exons of the UBE3A gene. These deletions are difficult to detect by conventional gene-scanning methods due to the masking effect by the non-delete…

Malecongenital hereditary and neonatal diseases and abnormalitiesClinical Biochemistrygene dosageBiochemistryGene dosageExonSettore BIO/13 - Biologia ApplicataAngelman syndromemedicineUBE3AHumansMultiplexGenetic TestingMultiplex ligation-dependent probe amplificationChildMolecular BiologyGeneticsbiologyubiquitin-protein ligasesgenetic association studiemedicine.diseaseMolecular biologyUniparental disomyUbiquitin ligaseAngelman syndromebiology.proteinMolecular MedicineOriginal ArticleFemaleGene Deletion
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The gene encoding ganglioside-induced differentiation-associated protein 1 is mutated in axonal Charcot-Marie-Tooth type 4A disease

2001

We identified three distinct mutations and six mutant alleles in GDAP1 in three families with axonal Charcot-Marie-Tooth (CMT) neuropathy and vocal cord paresis, which were previously linked to the CMT4A locus on chromosome 8q21.1. These results establish the molecular etiology of CMT4A (MIM 214400) and suggest that it may be associated with both axonal and demyelinating phenotypes.

Malecongenital hereditary and neonatal diseases and abnormalitiesDNA Mutational AnalysisMolecular Sequence DataMutantMutation MissenseNeural ConductionGenes RecessiveNerve Tissue ProteinsLocus (genetics)BiologyPolymerase Chain ReactionFrameshift mutationCharcot-Marie-Tooth DiseaseGeneticsHumansMissense mutationAge of OnsetAlleleChildFrameshift MutationGeneAllelesGeneticsBrainInfantExonsAnatomyPhenotypeAxonsPedigreeAmino Acid SubstitutionHaplotypesSpinal CordCodon NonsenseSpainChild PreschoolFemaleLod ScoreVocal cord paresisChromosomes Human Pair 8Demyelinating DiseasesNature Genetics
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Comparative multiplex dosage analysis in spinocerebellar ataxia type 2 patients.

2013

We developed a new application of comparative multiplex dosage analysis (CMDA) for evaluation of the ataxin 2 gene. Expansions of the triplet CAG can cause spinocerebellar ataxia type 2 (SCA2), a neurodegenerative disease with an autosomal-dominant mode of inheritance. Molecular diagnosis of SCA2 is routinely based on the use of conventional PCR to detect the CAG expansion. However, PCR does not amplify an allele with an expansion of many triplets (>80), which is typically found in infantile and juvenile forms of SCA2, thus leading to false negatives. We propose the analysis of the ATXN2 gene by CMDA to complement existing methods currently used for the detection of large expansions of the …

Malecongenital hereditary and neonatal diseases and abnormalitiesGenotypeGene DosagePrenatal diagnosisNerve Tissue ProteinsDiseaseAtaxin 2 Spinocerebellar ataxia type 2 Quantitative PCR Autosomal dominant Prenatal diagnosisSettore BIO/13 - Biologia ApplicataGeneticsMedicineHumansSpinocerebellar AtaxiasMultiplexAlleleMolecular BiologyGeneAllelesGeneticsbusiness.industryGeneral Medicinemedicine.diseaseReal-time polymerase chain reactionAtaxinsAtaxinCase-Control StudiesSpinocerebellar ataxiaFemalebusinessTrinucleotide Repeat ExpansionMultiplex Polymerase Chain ReactionGenetics and molecular research : GMR
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A novel mutation of the DHCR7 gene in a sicilian compound heterozygote with Smith-Lemli-Opitz Syndrome

2005

Introduction: Smith-Lemli-Opitz syndrome (SLOS) is an autosomal recessive disorder of cholesterol biosynthesis, resulting from deficient 7-dehydrocholesterol reductase (3β-hydroxysterol Δ7-reductase) activity, the enzyme responsible for conversion of 7-dehydrocholesterol to cholesterol. SLOS is most common among people of European descent, with a reported incidence of 1 per 20 000–60 000 newborns, depending on the diagnostic criteria and the reference population. More than 80 different mutations have been identified in several hundred patients. In Italy, SLOS appears to be a rare condition, probably because of underdiagnosis. Method: We analyzed by direct sequencing the 7-dehydrocholesterol…

Malecongenital hereditary and neonatal diseases and abnormalitiesHeterozygoteOxidoreductases Acting on CH-CH Group DonorsMutation MissenseBiologyReductaseCompound heterozygosityExonmedicineMissense mutationHumansGeneSicilyGeneticsnutritional and metabolic diseasesInfantGeneral Medicinemedicine.diseaseHuman geneticsPedigreeSmith-Lemli-Opitz SyndromeOxidoreductases Acting on CH-CH Group DonorSmith–Lemli–Opitz syndromeMutation (genetic algorithm)Human
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