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showing 10 items of 10618 documents

The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome)

2010

Mucopolysaccharidosis I Hurler (MPS IH) is a progressive multisystemic disorder caused by alpha-L-iduronidase deficiency. First choice of treatment in MPS IH children is haematopoietic stem cell transplantation (HSCT). The effect of HSCT has been shown to have limited influence on skeletal manifestations by poor penetration of musculoskeletal tissues by the enzyme derived from donor leucocytes. Aim of this study was to investigate the effect of HSCT on the craniocervical junction (CCJ) in Hurler patients. We analysed retrospectively sequential magnetic resonance imaging (MRI) scans of 30 patients with Hurler disease treated by HSCT since 1982 at the Royal Manchester Children's Hospital, UK,…

AdultMalemedicine.medical_specialtyAdolescentMucopolysaccharidosis Imedicine.medical_treatmentHematopoietic stem cell transplantationCohort StudiesYoung AdultMucopolysaccharidosis type IMucopolysaccharidosis IGeneticsmedicineHumansChildHurler syndromeGenetics (clinical)Retrospective StudiesBone Diseases Developmentalbusiness.industryOdontoid HypoplasiaSkullHematopoietic Stem Cell TransplantationBrainmedicine.diseaseMagnetic Resonance ImagingHypoplasiaSurgeryRadiographyTransplantationTreatment OutcomeGraft-versus-host diseaseChild PreschoolFemalebusinessJournal of Inherited Metabolic Disease
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Kallikrein–kinin system and fibrinolysis in hereditary angioedema due to factor XII gene mutation Thr309Lys

2009

In a subgroup of hereditary angioedema (HAE) patients with normal C1-esterase inhibitor levels, HAE is caused by a Thr309Lys mutation in the coagulation factor XII (F12) gene. The aim of this study was to examine elements of the kallikrein-kinin system ('contact system') and the downstream-linked coagulation, complement and fibrinolytic systems in the plasma of six patients with HAE caused by the Thr309Lys mutation and healthy probands. Blood samples were taken from participants during the symptom-free interval between attacks. Samples were analyzed for activity and concentrations of components of the kallikrein-kinin system and linked enzyme systems. The mean FXII clotting activity was 90%…

AdultMalemedicine.medical_specialtyAdolescentMutation MissenseKininsCoagulation Factor XIIFactor XIIaGene mutationYoung AdultInternal medicinemedicineHumansPoint MutationHereditary Angioedema Type IIIComplement Pathway ClassicalAgedAged 80 and overFactor XIIAngioedemaChemistryFibrinolysisDextran SulfateAngioedemas HereditaryPrekallikreinPrekallikreinBlood ProteinsHematologyGeneral MedicineMiddle AgedSilicon Dioxidemedicine.diseaseEnzyme ActivationEndocrinologyAmino Acid SubstitutionChromogenic CompoundsCoagulationTissue Plasminogen ActivatorHereditary angioedemaImmunologyFemaleKallikreinsmedicine.symptomcirculatory and respiratory physiologyBlood Coagulation & Fibrinolysis
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Simultaneous assessment of endothelial function, nitric oxide synthase activity, nitric oxide-mediated signaling, and oxidative stress in individuals…

2007

Abstract Background: Endothelial function is impaired in hypercholesterolemia and atherosclerosis. Based on mostly indirect evidence, this impairment is attributed to reduced synthesis or impaired biological activity of endothelium-derived nitric oxide (NO). It was the aim of this study to directly estimate and compare whole-body NO production in normo- and hypercholesterolemia by applying a nonradioactive stable isotope dilution technique in vivo. Methods: We enrolled 12 normocholesterolemic and 24 hypercholesterolemic volunteers who were all clinically healthy. To assess whole-body NO synthesis, we intravenously administered l-[guanidino-(15N2)]-arginine and determined the urinary excreti…

AdultMalemedicine.medical_specialtyAdolescentNitric Oxide Synthase Type IIIClinical BiochemistryHypercholesterolemiaVasodilationEndothelial NOSArginineDinoprostNitric OxideNitric oxideExcretionchemistry.chemical_compoundSex FactorsInternal medicinemedicineHumansCyclic GMPAgedNitratesbiologyBiochemistry (medical)Nitric Oxide Synthase Type IIIMiddle AgedNitric oxide synthaseOxidative StressEndocrinologychemistrybiology.proteinFemaleEndothelium VascularNitric Oxide SynthaseAsymmetric dimethylarginineLipoproteinClinical chemistry
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Association between polymorphisms of endothelial nitric oxide synthase gene (NOS3) and left posterior wall thickness (LPWT) of the heart in Fabry dis…

2008

Fabry disease is an X-chromosomal storage disorder due to loss-of-function mutations of the GLA gene encoding the lysosomal enzyme α-galactosidase A. Accumulating glycosphingolipid deposits disturb the function of various cells, in particular that of myocytes, arterial smooth-muscle cells, and vascular endothelium. Hypertrophic cardiomyopathy, for example measured by left posterior wall thickness (LPWT) of the heart, represents a major component of Fabry disease morbidity in adult patients. Endothelium-derived nitric oxide (eNO), produced by eNO synthase (eNOS), is a key regulator of vessel wall function and cardiovascular homeostasis. We analysed the effect of the polymorphisms c.894G > T …

AdultMalemedicine.medical_specialtyAdolescentNitric Oxide Synthase Type IIIMutation MissenseMinisatellite RepeatsYoung AdultSex FactorsGene FrequencyInternal medicineGermanyGenotypeGeneticsMedicineHumansGenetic Predisposition to DiseaseAlleleChildAllele frequencyGenetics (clinical)AgedUltrasonographyGeneticsAlpha-galactosidasePolymorphism Geneticbiologybusiness.industryMyocardiumHaplotypeHypertrophic cardiomyopathyNitric Oxide Synthase Type IIIExonsCardiomyopathy HypertrophicMiddle Agedmedicine.diseaseFabry diseaseIntronsEndocrinologyPhenotypeHaplotypesCase-Control Studiesalpha-Galactosidasebiology.proteinFabry DiseaseRegression AnalysisFemaleHypertrophy Left VentricularbusinessJournal of inherited metabolic disease
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Fissurectomy combined with anoplasty and injection of botulinum toxin in treatment of anterior chronic anal fissure with hypertonia of internal anal …

2010

Abstract BACKGROUND: In patients affected by anterior chronic anal fissure (CAAF) with hypertonia of the internal anal sphincter (IAS), the role of IAS hypertonia remains unclear. The aim of this study was to evaluate the efficacy of fissurectomy combined with advancement flap and IAS injection of botulinum toxin in healing the CAAF with hypertonia of IAS resistant to medical therapy. METHODS: Ten consecutive patients were enrolled. Anorectal manometry was performed preoperatively and at 6 months. CAAF with hypertonia was defined as those associated with maximum resting pressure (MRP) values higher than 85 mmHg. All patients underwent fissurectomy and anoplasty with advancement skin flap co…

AdultMalemedicine.medical_specialtyAdolescentPilot ProjectsInjections IntramuscularSurgical FlapsInternal anal sphincterCohort StudiesYoung AdultMuscle HypertoniamedicineHumanschronic anal fissureBotulinum Toxins Type ADefecationbusiness.industryAnorectal manometryGastroenterologyRecovery of FunctionMiddle AgedBotulinum toxinColorectal surgerySurgeryClinical trialTreatment OutcomeNeuromuscular AgentsAnesthesiaAnterior chronic anal fissure Fissurectomy Advancement flap Botulinum toxin.HypertoniaDefecationSurgeryFemaleFissure in Anomedicine.symptombusinessmedicine.drugAbdominal surgeryTechniques in coloproctology
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Prevalence of plasma lipid abnormalities and its association with glucose metabolism in Spain: The di@bet.es study

2013

Abstract Introduction Dyslipidemia is a significant contributor to the elevated CVD risk observed in type 2 diabetes mellitus. We assessed the prevalence of dyslipidemia and its association with glucose metabolism status in a representative sample of the adult population in Spain and the percentage of subjects at guideline-recommended LDL-C goals. Material and methods The di@bet.es study is a national, cross-sectional population-based survey of 5728 adults. Results A total of 4776 subjects were studied. Dyslipidemia was diagnosed in 56.8% of subjects; only 13.2% of subjects were treated with lipid lowering drugs. Lipid abnormalities were found in 56.8% of Spanish adults: 23.3% with high LDL…

AdultMalemedicine.medical_specialtyAdolescentPopulationAdult populationCarbohydrate metabolismPrediabetic StateYoung AdultRisk FactorsInternal medicinePlasma lipidsPrevalencemedicineHumansPharmacology (medical)educationAgedDyslipidemiasHypolipidemic Agentseducation.field_of_studybusiness.industryCholesterol HDLType 2 Diabetes MellitusCholesterol LDLMiddle Agedmedicine.diseaseSpanish populationCross-Sectional StudiesGlucoseEndocrinologyDiabetes Mellitus Type 2Cardiovascular DiseasesSpainFemalelipids (amino acids peptides and proteins)Lipid loweringCardiology and Cardiovascular MedicinebusinessDyslipidemiaClínica e Investigación en Arteriosclerosis
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How ineffective hypertension control in subjects treated with angiotensin-converting enzyme inhibitors is related to polymorphisms in the renin-angio…

2009

Abstract Purpose To investigate how genetic polymorphisms of the renin-angiotensin-aldosterone system (RAAS) influence hypertension (HT) control with angiotensin-converting enzyme inhibitor drugs (ACEI). Methods A case–control, cross-sectional population-based nested study (n = 1514) included hypertensive patients treated with ACEI drugs, either alone or with other antihypertensive drugs. We differentiated between those who did not control their HT (cases) with those who did (controls). Each group's characteristics were compared to determine the risk of non-controlled HT associated with RAAS polymorphisms by adjusting for different variables. Results rs11571074 obtained an ORa of 5.26 for T…

AdultMalemedicine.medical_specialtyAdolescentPopulationPharmaceutical ScienceAngiotensin-Converting Enzyme InhibitorsPharmacologyYoung AdultPolymorphism (computer science)Internal medicineRenin–angiotensin systemGenotypemedicineHumanseducationAgedAged 80 and overeducation.field_of_studyPolymorphism GeneticHypertension controlbiologybusiness.industryCase-control studyAngiotensin-converting enzymeMiddle AgedEndocrinologyCross-Sectional StudiesCase-Control StudiesACE inhibitorHypertensionbiology.proteinFemalebusinessmedicine.drugEuropean journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences
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Central nervous system involvement in late-onset Pompe disease: clues from neuroimaging and neuropsychological analysis

2018

Background and purpose Late-onset Pompe disease (LOPD) is a rare, multisystem disorder that is well established to mainly impair skeletal muscle function. Systematic studies exploring brain functions in LOPD are lacking. The aim of this study was to detect morphological and functional brain alterations as well as neuropsychological impairment in LOPD. Methods We studied 21 patients (10 male, mean age 49 ± 18.4 years) with defined diagnosis of LOPD, divided into two groups: one with pre-symptomatic hyperCKemia with no muscle weakness and the second with limb-girdle muscle weakness. All patients underwent 3T magnetic resonance imaging (MRI) to obtain morphological/angiographic evaluation as w…

AdultMalemedicine.medical_specialtyAdolescentSmoker scoreNeuropsychological Testscerebrovascular abnormalitieslate-onset Pompe diseaseYoung Adult03 medical and health sciences0302 clinical medicineAtrophyNeuroimagingInternal medicineConnectomemedicinecerebrovascular abnormalities Fazekas score functional magnetic resonance imaging late-onset Pompe disease Pompe disease Smoker score Neurology Neurology (clinical)HumansCognitive Dysfunction030212 general & internal medicineNeuropsychological assessmentAge of OnsetGray MatterAgedmedicine.diagnostic_testGlycogen Storage Disease Type IIbusiness.industryMuscle weaknessPompe diseaseMagnetic resonance imagingMiddle Agedmedicine.diseaseMagnetic Resonance Imagingfunctional magnetic resonance imagingHyperintensityFazekas scoreSuperior frontal gyrusNeurologyBrain sizeCardiologyFemaleNeurology (clinical)medicine.symptombusiness030217 neurology & neurosurgery
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Increased sensitivity of the neuronal nicotinic receptor alpha-2 subunit causes familial epilepsy with nocturnal wandering and ictal fear

2006

Sleep has traditionally been recognized as a precipitating factor for some forms of epilepsy, although differential diagnosis between some seizure types and parasomnias may be difficult. Autosomal dominant frontal lobe epilepsy is characterized by nocturnal seizures with hyperkinetic automatisms and poorly organized stereotyped movements and has been associated with mutations of the α4 and β2 subunits of the neuronal nicotinic acetylcholine receptor. We performed a clinical and molecular genetic study of a large pedigree segregating sleep-related epilepsy in which seizures are associated with fear sensation, tongue movements, and nocturnal wandering, closely resembling nightmares and sleep …

AdultMalemedicine.medical_specialtyAdolescentSomnambulismMolecular Sequence DataMutation MissenseAutosomal dominant nocturnal frontal lobe epilepsyReceptors NicotinicBiologymedicine.disease_causeLigandsNicotinicArticleEpilepsyBIO/09 - FISIOLOGIAInternal medicineAcetylcholine; Adolescent; Adult; Aged; Aged 80 and over; Amino Acid Sequence; Epilepsy; Female; Humans; Ligands; Male; Molecular Sequence Data; Mutation Missense; Neurons; Pedigree; Receptors Nicotinic; Somnambulism; FearReceptorsmedicine80 and overGeneticsHumansIctalGenetics(clinical)Amino Acid SequenceGenetics (clinical)Acetylcholine receptorAgedAged 80 and overNeuronsMutationEpilepsySeizure typesFearmedicine.diseaseAcetylcholinePedigreeNicotinic acetylcholine receptorNicotinic agonistEndocrinologyMutationnAChR patch-clamp ADNFLE sleep-related epilepsy M1 TM1 ACh nicotineSettore MED/26 - NeurologiaFemaleMissense
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Global and regional cortical thinning in first-episode psychosis patients: relationships with clinical and cognitive features

2010

BackgroundThe thickness of the cortical mantle is a sensitive measure for identifying alterations in cortical structure. We aimed to explore whether first episode schizophrenia patients already show a significant cortical thinning and whether cortical thickness anomalies may significantly influence clinical and cognitive features.MethodWe investigated regional changes in cortical thickness in a large and heterogeneous sample of schizophrenia spectrum patients (n=142) at their first break of the illness and healthy controls (n=83). Magnetic resonance imaging brain scans (1.5 T) were obtained and images were analyzed by using brains2. The contribution of sociodemographic, cognitive and clinic…

AdultMalemedicine.medical_specialtyAdolescentbrainBrain mappingArticleYoung AdultCognitionCortex (anatomy)Internal medicineImage Processing Computer-AssistedmedicineHumansApplied PsychologyCerebral CortexPsychiatric Status Rating ScalesAnalysis of VarianceBrain Mappingmedicine.diagnostic_testAge FactorsMagnetic resonance imagingMiddle Agedcortical thicknessmedicine.diseaseMagnetic Resonance ImagingendophenotypeschizophreniaPsychiatry and Mental healthmedicine.anatomical_structureCerebral cortexSchizophreniaEndophenotypeCardiologyFemaleAnalysis of varianceAge of onsetPsychologyNeuroscienceMRIPsychological Medicine
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