Search results for "RICS"

showing 10 items of 14086 documents

The role of registries in rare genetic lipid disorders: Review and introduction of the first global registry in lipoprotein lipase deficiency

2017

International audience; A good understanding of the natural history of rare genetic lipid disorders is a pre-requisite for successful patient management. Disease registries have been helpful in this regard. Lipoprotein Lipase Deficiency (LPLD) is a rare, autosomal-recessive lipid disorder characterized by severe hypertriglyceridemia and a very high risk for recurrent acute pancreatitis, however, only limited data are available on its natural course. Alipogene tiparvovec (Glybera (R)) is the first gene therapy to receive Marketing Authorization in the European Union; GENIALL (GENetherapy In the MAnagement of Lipoprotein Lipase Deficiency), a 15-year registry focusing on LPLD was launched in …

0301 basic medicinePediatricsPathologySettore MED/09 - Medicina Interna[SDV]Life Sciences [q-bio]Familial hypercholesterolemiaDisease030204 cardiovascular system & hematologyGeneTHERAPY0302 clinical medicineFamilialRisk FactorsHyperchylomicronemiaAlipogene tiparvovecRegistriesFAMILIAL HYPERCHOLESTEROLEMIAmedia_commonHypertriglyceridemiaPrognosis3. Good healthNatural historySystematic reviewPhenotypeDISEASESSAFETYHyperlipoproteinemia Type ICardiology and Cardiovascular Medicinemedicine.medical_specialtyAPHERESISRegistryFamilial chylomicronemia syndromeGENIALLLysosomal acid lipase deficiencyLipid Metabolism Inborn Errors03 medical and health sciencesLipoprotein lipase deficiencyRare DiseasesGene therapychylomicronemia syndromemedicinemedia_common.cataloged_instanceHumansGenetic Predisposition to DiseaseEuropean unionLipoprotein lipase deficiency (LPLD)business.industryALIPOGENE TIPARVOVEC AAV1-LPLS447Xmedicine.diseaseAlipogene tiparvovecLipoprotein Lipase030104 developmental biologyOrphan diseasebusiness
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Postpartum listeria meningitis

2020

Highlights • Listeria monocytogenes is a rare cause of meningitis in immunocompetent patient. • Pregnancy and puerperium are accompanied by a decrease in cellular immunity. • Pregnancy and puerperium should be considered as risk factors for invasive listeriosis.

0301 basic medicinePediatricsmedicine.medical_specialty030106 microbiologyInfectious and parasitic diseasesRC109-216DiseaseAbortionmedicine.disease_causeAsymptomaticArticle03 medical and health sciences0302 clinical medicineImmune systemListeria monocytogenesmedicineMeningitis030212 general & internal medicinereproductive and urinary physiologyListeria monocytogenePregnancybusiness.industryPuerperiummedicine.diseaseAntimicrobialListeria monocytogenesfemale genital diseases and pregnancy complicationsMeningitiInfectious Diseasesmedicine.symptombusinessMeningitisIDCases
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11 and 15-month-old infants do not compensate immediately for energy variation, and no further adjustment occurs 12 or 24 hours later

2021

International audience; Previously, we demonstrated that, in the short term, infants undercompensated for the energy from a preload given 25 min before an ad libitum meal. However, although not consistent, there is evidence in young children that caloric adjustment may occur over longer periods. We investigated the extent to which further energy adjustment occurs up to 24 h after a single meal preceded by preloads of varying energy density (ED) in infants that are 11 and 15 months old. Short-term caloric adjustment was measured in 11- and 15-month-old infants through a preload paradigm meal in the laboratory. To assess their caloric adjustment over longer periods (12 and 24 h), we used 24 h…

0301 basic medicinePediatricsmedicine.medical_specialtyAppetite controlCOMPXEnergy balanceAppetite030209 endocrinology & metabolism03 medical and health sciences0302 clinical medicineappetite controlpreload paradigmmedicineHumansChildMealsGeneral PsychologyMeal[SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/Pediatrics030109 nutrition & dieteticsNutrition and DieteticsAppetite Regulationbusiness.industrydigestive oral and skin physiologyInfantCaloric theoryDiet RecordsPreloadChild Preschooldietary recordEnergy densityEnergy variationenergy intakecaloric adjustmentmedicine.symptomEnergy MetabolismbusinessWeight gain[SDV.AEN]Life Sciences [q-bio]/Food and Nutrition
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EAST/SeSAME syndrome: Review of the literature and introduction of four new Latvian patients.

2018

EAST (Epilepsy, Ataxia, Sensorineural deafness, Tubulopathy) or SeSAME (Seizures, Sensorineural deafness, Ataxia, Mental retardation, and Electrolyte imbalance) syndrome is a rare autosomal recessive syndrome first described in 2009 independently by Bockenhauer and Scholl. It is caused by mutations in KCNJ10, which encodes Kir4.1, an inwardly rectifying K+ channel found in the brain, inner ear, kidney and eye. To date, 16 mutations and at least 28 patients have been reported. In this paper, we review mutations causing EAST/SeSAME syndrome, clinical manifestations in detail, and efficacy of treatment in previously reported patients. We also report a new Latvian kindred with 4 patients. In co…

0301 basic medicinePediatricsmedicine.medical_specialtyAtaxiaHearing Loss SensorineuralKCNJ10030105 genetics & hereditySensorineural deafnessKidney03 medical and health sciencesEpilepsyTubulopathySeizuresIntellectual DisabilityIntellectual disabilityGeneticsmedicineEAST syndromeHumansEye AbnormalitiesPotassium Channels Inwardly RectifyingGenetics (clinical)SeSAME syndromebiologybusiness.industryBrainmedicine.diseaseLatvia030104 developmental biologyPhenotypeEar InnerMutationbiology.proteinmedicine.symptombusinessClinical genetics
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Maternal Diet and Nutrient Requirements in Pregnancy and Breastfeeding. An Italian Consensus Document.

2016

The importance of lifestyle and dietary habits during pregnancy and breastfeeding, for health of mothers and their offspring, is widely supported by the most recent scientific literature. The consumption of a varied and balanced diet from the preconceptional period is essential to ensure both maternal well-being and pregnancy outcomes. However, the risk of inadequate intakes of specific micronutrients in pregnancy and lactation is high even in the most industrialized countries. This particularly applies to docosahexaenoic acid (DHA), iron, iodine, calcium, folic acid, and vitamin D, also in the Italian population. Moreover, the risk of not reaching the adequate nutrient supply is increased …

0301 basic medicinePediatricsmedicine.medical_specialtyConsensusOffspringbreastfeedingBreastfeedingMotherslcsh:TX341-641ReviewOverweight03 medical and health sciencesEnvironmental healthHumansMedicinemicronutrientPregnancynutrient requirement030109 nutrition & dieteticsNutrition and Dieteticsbusiness.industryNutritional RequirementsFeeding BehaviorMaternal Nutritional Physiological Phenomenaadequate intakemedicine.diseaseMicronutrientDietDHABreast FeedingHuman nutritionItalyDietary Reference IntakemicronutrientsFemalepregnancymedicine.symptomUnderweightbusinesslcsh:Nutrition. Foods and food supplyFood Science
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Neoplasia mandibular y gastrostomía: a propósito de un caso nutricional

2018

Se expone el caso de un varón de 47 años con carcinoma epidermoide mandibular. Su evolución ocasiona la aparición de una desnutrición calórica severa que requiere una valoración nutricional completa con el método SENPE-SEDOM. Dicha situación se agrava con el tratamiento quirúrgico y oncológico, desarrollando una disfagia mecánica grave, valorada y diagnosticada con el Método de Exploración Clínica Volumen-Viscosidad (MECV-V). El paciente requiere progresivas adaptaciones dietéticas tanto de textura como de aporte nutricional, precisando el uso de sonda de alimentación por gastrostomía y de nutrición enteral a través de ella. La finalidad es mejorar el estado nutricional, que había empeorado…

0301 basic medicinePediatricsmedicine.medical_specialtyDesnutriciónRC620-627Neoplasias de Cabeza y CuelloDiseaseEnteral administrationGastrostomy feeding tube03 medical and health sciencesTrastornos de DegluciónmedicineBasal cellTX341-641GastrostomíaNutritional diseases. Deficiency diseasesNutrición Enteral030109 nutrition & dieteticsNutrition and Dieteticsbusiness.industryNutrition. Foods and food supplymedicine.diseaseDysphagiaDietoterapia.MalnutritionParenteral nutritionDietoterapiamedicine.symptombusinessOral feedingFood Science
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Quality of Life in Individuals with Diabetic Foot Syndrome.

2020

Background and Objective: Diabetic foot syndrome (DFS) is a common long-term complication of diabetes mellitus. DFS has recently been associated with adverse effects that could further impair the quality of life of diabetic patients, and increase the social and economic burden, morbidity, and premature mortality of the disease. The main physio-pathological basis of DFS is due to diabetesinduced neuropathy and angiopathy in the lower limbs and feet. Patients diagnosed with DFS must significantly modify their daily habits in order to cope with signs and symptoms of DFS and this can alter their quality of life. The objective of this review is to summarize the evidence regarding the economic, …

0301 basic medicinePediatricsmedicine.medical_specialtyEndocrinology Diabetes and Metabolism030209 endocrinology & metabolismDiseaseAmputation SurgicalAngiopathy03 medical and health sciences0302 clinical medicineQuality of lifeDiabetes mellitusSurveys and QuestionnairesDiabetes MellitusImmunology and AllergyMedicineHumansAdverse effectDepression (differential diagnoses)business.industrymedicine.diseaseDiabetic footDiabetic Foot030104 developmental biologySocioeconomic FactorsQuality of LifeHealth educationbusinessEndocrine, metabolicimmune disorders drug targets
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Autoimmune polyglandular diseases.

2019

Autoimmune polyglandular diseases (APD) are defined as the presence of two autoimmune -induced endocrine failures. With respect to the significant morbidity and potential mortality of APD, the diagnostic objective is to detect APD at an early stage, with the advantage of less frequent complications, effective therapy and better prognosis. This requires that patients at risk be regularly screened for subclinical endocrinopathies prior to clinical manifestation. Regarding the time interval between manifestation of first and further endocrinopathies, regular and long-term follow-up is warranted. Quality of life and psychosocial status are poor in APD patients and involved relatives. Familial c…

0301 basic medicinePediatricsmedicine.medical_specialtyEndocrinology Diabetes and Metabolism030209 endocrinology & metabolismFamilial clusteringClinical manifestationEndocrine System DiseasesAutoimmune Diseases03 medical and health sciences0302 clinical medicineEndocrinologyQuality of lifeMedicineHumansIn patientStage (cooking)Polyendocrinopathies AutoimmuneSubclinical infectionPatient Care Teambusiness.industrymusculoskeletal neural and ocular physiologyIncidence030104 developmental biologycardiovascular systemQuality of LifeInterdisciplinary CommunicationHigh incidenceMorbiditybusinessPsychosocialcirculatory and respiratory physiologyBest practiceresearch. Clinical endocrinologymetabolism
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Incidence, Diagnosis, and Outcome of Acquired Thrombotic Thrombocytopenic Purpura (aTTP): A Nationwide Survey By the Spanish Apheresis Group

2019

Introduction: Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disease characterized by a severe deficiency of the enzymatic activity of ADAMTS13 caused by autoantibodies, with an incidence of 3-4 x106inhabitants per year according to the few published data available. Accurate estimates of the incidence of aTTP are important to assess the resources required for current treatments and to anticipate the need to develop new treatments. The aim of this study was to determine the actualincidence of aTTP in Spain, as well as its diagnosis, management, and associated complications. Material and methods:A cross-sectional surveywascarried out among hematologists working in Spanish hospi…

0301 basic medicinePediatricsmedicine.medical_specialtyExacerbationImmunologyPopulationThrombotic thrombocytopenic purpuraBiochemistrylaw.invention03 medical and health sciences0302 clinical medicineInterquartile rangelawmedicineeducationeducation.field_of_studybusiness.industryIncidence (epidemiology)Mortality rateCell BiologyHematologymedicine.diseaseIntensive care unit030104 developmental biologyRituximabbusiness030215 immunologymedicine.drugBlood
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Jacobsen syndrome and neonatal bleeding: report on two unrelated patients

2021

Abstract Introduction In 1973, Petrea Jacobsen described the first patient showing dysmorphic features, developmental delay and congenital heart disease (atrial and ventricular septal defect) associated to a 11q deletion, inherited from the father. Since then, more than 200 patients have been reported, and the chromosomal critical region responsible for this contiguous gene disorder has been identified. Patients’ presentation We report on two unrelated newborns observed in Italy affected by Jacobsen syndrome (JBS, also known as 11q23 deletion). Both patients presented prenatal and postnatal bleeding, growth and developmental delay, craniofacial dysmorphisms, multiple congenital anomalies, a…

0301 basic medicinePediatricsmedicine.medical_specialtyGenotype-phenotype correlationHeart diseaseGenetic counselingCase ReportIn situ hybridization030105 genetics & heredityPediatricsRJ1-57003 medical and health sciences0302 clinical medicineaCGHJBSmedicineHumansJacobsen Distal 11q Deletion SyndromeJacobsen syndromeCraniofacialGenetic Association StudiesCerebral Hemorrhage11q23 deletionbusiness.industryInfant NewbornEarly diagnosimedicine.diseaseEarly diagnosisPancytopeniaThrombocytopeniaItalyFemalePresentation (obstetrics)business030217 neurology & neurosurgeryComparative genomic hybridizationItalian Journal of Pediatrics
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