Search results for "SAC"

showing 10 items of 3337 documents

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

2006

Purpose: To evaluate the safety and efficacy of recombinant human iduronate-2-sulfatase (idursulfase) in the treatment of mucopolysaccharidosis II. Methods: Ninety-six mucopolysaccharidosis II patients between 5 and 31 years of age were enrolled in a double-blind, placebo-controlled trial. Patients were randomized to placebo infusions, weekly idursulfase (0.5 mg/kg) infusions or every-other-week infusions of idursulfase (0.5 mg/kg). Efficacy was evaluated using a composite endpoint consisting of distance walked in 6 minutes and the percentage of predicted forced vital capacity based on the sum of the ranks of change from baseline. Results: Patients in the weekly and every-other-week idursul…

AdultMaleVital capacitymedicine.medical_specialtyAdolescentIdursulfaseVital CapacityIduronate SulfatasePlacebolaw.inventionchemistry.chemical_compoundDouble-Blind MethodRandomized controlled trialElosulfase alfalawInternal medicinemedicineHumansMucopolysaccharidosis type IIChildGenetics (clinical)GlycoproteinsMucopolysaccharidosis IIbusiness.industryHunter syndromeDrug ToleranceEnzyme replacement therapymedicine.diseaseRecombinant ProteinsSurgerychemistryChild PreschoolSafetybusinessmedicine.drugGenetics in Medicine
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Sacral spina bifida occulta rare occurrence in Byzantine Belentepe population in Muğla, Turkey: A possible case for adequate folic acid intake.

2018

Spina bifida may occur during the first weeks after conception; folic acid deficiency is strongly related to this anomaly. We argue that the low prevalence rate of spina bifida may indicate a relatively good nutrition state of a population, given that folic acid is found in many food products commonly eaten. The aim of this study is to examine the relationship between folic acid intake and spina bifida occulta prevalence in the Belentepe Byzantine population in Anatolian peninsula, and to compare the prevalence rates with various other ancient Anatolian populations by focusing on sacral spina bifida occulta in the Byzantine population. A total of 62 available human sacra were included in th…

AdultMalecongenital hereditary and neonatal diseases and abnormalitiesSacrumAdolescentTurkeyPopulationPrevalenceNutritional StatusBiologyFolic Acid DeficiencySpina Bifida OccultaAnthropology PhysicalSpina bifida occultaYoung AdultmedicineHumanseducationChildHistory Ancienteducation.field_of_studySpina bifidaInfant NewbornInfantNutritional statusMiddle Agedmedicine.diseaseHistory Medievalnervous system diseasesFolic acidAnthropologyFood productsChild PreschoolFolic acid intakeFemaleByzantiumDemographyHomo : internationale Zeitschrift fur die vergleichende Forschung am Menschen
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Abnormal Somatosensory Evoked Potentials Indicate Compressive Cervical Myelopathy in Mucopolysaccharidoses

2000

Compressive myelopathy at the cranio-cervical junction is a complication of mucopolysaccharidoses (MPS). To detect cervical myelopathy we recorded median and posterior tibial nerve SEPs in 15 patients aged 2.4 - 33.4 years (median 8.8 years) with MPS I-S (n = 3), MPS IVA (n = 8) and MPS VI (n = 4). In addition to the cortical waveforms we recorded the subcortical median nerve SEPs N13b and P13 generated near the cranio-cervical junction and the lemniscal P30 after posterior tibial nerve stimulation. MRI studies in 13 subjects revealed spinal cord compression at the cranio-cervical junction in 10 patients; 5 patients had an increased signal intensity on the T2-weighted initial MRI indicating…

AdultMalecongenital hereditary and neonatal diseases and abnormalitiesmedicine.medical_specialtyAdolescentSensitivity and SpecificityCentral nervous system diseaseMyelopathySpinal cord compressionEvoked Potentials SomatosensorymedicineHumansChildbusiness.industryGeneral MedicineCervical cord compressionMucopolysaccharidosesmedicine.diseaseSpinal cordMagnetic Resonance ImagingMedian nerveMedian NerveSurgerybody regionsmedicine.anatomical_structureSpinal CordSomatosensory evoked potentialChild PreschoolPediatrics Perinatology and Child HealthFemaleNeurology (clinical)RadiologyTibial NervebusinessSpinal Cord CompressionMyelomalaciaNeckNeuropediatrics
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Acquired monocular elevation paresis. An asymmetric upgaze palsy.

1992

Five patients with acquired monocular elevation paresis were investigated using direct current electrooculography. With recovery, upward saccade velocities significantly increased in both eyes in all patients. The gain of upward-following eye movements significantly increased in the paretic eye of all patients and in the opposite eye of four patients. These findings are interpreted in terms of an asymmetric upgaze palsy which clinically presented as monocular elevation paresis in the more severely affected eye. A brainstem lesion contralateral to monocular elevation paresis was suggested in four patients by contralateral Horner's syndrome and contralateral abduction paresis, each in one pat…

AdultMalegenetic structuresEye DiseasesEye MovementsEye diseaseHorner syndromePtosisUpgaze palsymedicineParalysisSaccadesHumansParalysisParesisAgedOphthalmoplegiamedicine.diagnostic_testbusiness.industryElectrooculographyMiddle Agedmedicine.diseaseeye diseasesAnesthesiaSaccadeFemalesense organsNeurology (clinical)medicine.symptombusinessBrain : a journal of neurology
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Functional Magnetic Resonance Imaging Activations of Cortical Eye Fields during Saccades, Smooth Pursuit, and Optokinetic Nystagmus

2009

Saccades, smooth pursuit, and optokinetic nystagmus (OKN) are three basic eye movements in our ocular motor repertoire that enable us to explore the visual field. These eye movements are cortically controlled in different cortical eye fields, including the frontal eye fields (FEF) and parietal eye fields (PEF), as well as the motion-sensitive visual area MT+/V5. It is not known if this cortical control is organized in parallel cortico-cortical networks or in adjacent subregions of one system. Nor do we know where the specific eye fields are exactly located. Functional magnetic resonance imaging (fMRI) was used to investigate these open questions about the FEF, PEF, and MT+/V5. Activations o…

AdultMalegenetic structuresGeneral Biochemistry Genetics and Molecular BiologySmooth pursuitHistory and Philosophy of ScienceCortex (anatomy)SaccadesmedicineHumansAgedmedicine.diagnostic_testGeneral NeuroscienceEye movementOptokinetic reflexMiddle AgedFrontal eye fieldsMagnetic Resonance Imagingeye diseasesParietal eyeVisual fieldmedicine.anatomical_structureFemalesense organsVisual FieldsFunctional magnetic resonance imagingPsychologyNeuroscienceAnnals of the New York Academy of Sciences
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Joint involvement in psoriasis.

1974

21 unselected hospital patients with psoriasis (7 female, 14 males) were examined for joint lesions by clinical, roentgenological and scintigraphical methods (6 mCi99mTc-Pertechnetate, 4 mCi99mTc-Pyrophosphate). According to traditional concepts only 5 patients suffered from psoriatic arthropathy, in 8 other patients an extensive case history revealed some joint complaints.

AdultMalemedicine.medical_specialtyAdolescentArthritisDermatologyArthritis RheumatoidDiagnosis DifferentialPsoriatic arthritisPsoriasisArthropathymedicineHumansPsoriasisHospital patientsChildRadionuclide ImagingAgedSacroiliac jointArthritis Infectiousbusiness.industryFootArthritisTechnetiumSacroiliac JointGeneral MedicineMiddle Agedmedicine.diseaseHandDermatologySurgerymedicine.anatomical_structureJoint involvementHalluxFemaleJoint DiseasesbusinessJoint lesionsArchiv fur dermatologische Forschung
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Utility of presepsin (sCD14-ST) as a diagnostic and prognostic marker of sepsis in the emergency department

2015

Presepsin (PSEP) is released during infectious diseases and can be detected in the blood. PSEP has shown promising results as sepsis marker. We examined the diagnostic and prognostic validity of PSEP in patients suspicious of sepsis on admission in the emergency department (ED). Methods One hundred twenty three patients with signs of SIRS and/or sepsis and 123 healthy individuals were enrolled. PSEP was determined on admission, after 8, 24 and 72 h. Results Mean PSEP concentrations of the control group and the patient group were 130 and 1945 pg/ml. PSEP differed between SIRS, sepsis, severe sepsis and septic shock and showed strong association with 30-day mortality ranging from 10.3% in the…

AdultMalemedicine.medical_specialtyAdolescentClinical BiochemistryLipopolysaccharide ReceptorsBiochemistrySepsisDiagnosis DifferentialYoung AdultInternal medicineSepsisDiagnosismedicineHumansYoung adultIntensive care medicineAPACHE IIbusiness.industrySeptic shockEmergency departmentBiochemistry (medical)PresepsinGeneral MedicineEmergency departmentOutcome predictionMiddle Agedmedicine.diseasePrognosisShock SepticPeptide FragmentsQuartileClose relationshipFemalebusinessOutcome predictionEmergency Service HospitalBiomarkers
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Effectiveness of vaccination with 23-valent pneumococcal polysaccharide vaccine in preventing hospitalization with laboratory confirmed influenza dur…

2013

Background: Since influenza predisposes to bacterial pneumonia caused by Streptococcus pneumoniae, studies have suggested that pneumococcal vaccination might reduce its occurrence during pandemics. We assessed the effectiveness of pneumococcal polysaccharide vaccination alone and in combination with influenza vaccination in preventing influenza hospitalization during the 2009–2010 pandemic wave and 2010–2011 influenza epidemic. Methods: We conducted a multicenter case-control study in 36 Spanish hospitals. We selected patients aged ≥ 18 y hospitalized with confirmed influenza and two hospitalized controls per case, matched according to age, date of hospitalization and province of residence.…

AdultMalemedicine.medical_specialtyAdolescentInfluenza vaccineImmunologymedicine.disease_causePneumococcal VaccinesYoung AdultInternal medicineStreptococcus pneumoniaePandemicInfluenza HumanmedicineImmunology and AllergyHumansYoung adultAgedPharmacologyAged 80 and overbusiness.industryBacterial pneumoniaCase-control studyvirus diseasesMiddle AgedPneumonia Pneumococcalmedicine.diseasePneumococcal polysaccharide vaccineVaccinationHospitalizationInfluenza VaccinesSpainCase-Control StudiesImmunologyCosts and Cost AnalysisFemalebusinessResearch Paper
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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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Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management

2011

The mucopolysaccharidoses (MPSs) are inherited lysosomal storage disorders caused by the absence of functional enzymes that contribute to the degradation of glycosaminoglycans (GAGs). The progressive systemic deposition of GAGs results in multi-organ system dysfunction that varies with the particular GAG deposited and the specific enzyme mutation(s) present. Cardiac involvement has been reported in all MPS syndromes and is a common and early feature, particularly for those with MPS I, II, and VI. Cardiac valve thickening, dysfunction (more severe for left-sided than for right-sided valves), and hypertrophy are commonly present; conduction abnormalities, coronary artery and other vascular in…

AdultMalemedicine.medical_specialtyAdolescentMucopolysaccharidosisClinical SciencesHeart Valve DiseasesReviewComorbidityCoronary Artery DiseaseDiseaseMuscle hypertrophyCoronary artery diseaseElectrocardiographyVentricular hypertrophyTachycardiaInternal medicineGeneticsmedicineHumansGenetics(clinical)Age of OnsetSinusChildPreschoolGenetics (clinical)GlycosaminoglycansGenetics & Hereditymedicine.diagnostic_testbusiness.industryMitral Valve InsufficiencyHypertrophyAortic Valve StenosisEnzyme replacement therapyMucopolysaccharidosesMiddle Agedmedicine.diseaseLeft VentricularCausalityTachycardia SinusEchocardiographyChild PreschoolAortic valve stenosisCardiologyHypertrophy Left VentricularFemalebusinessElectrocardiographyJournal of Inherited Metabolic Disease
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