Search results for " Hereditary"

showing 3 items of 3 documents

International BEAT-PCD Consensus Statement for Infection Prevention and Control for Primary Ciliary Dyskinesia in collaboration with ERN-LUNG PCD Cor…

2021

Introduction In primary ciliary dyskinesia (PCD) impaired mucociliary clearance leads to recurrent airway infections and progressive lung destruction, and concern over chronic airway infection and patient-to-patient transmission is considerable. So far, there has been no defined consensus on how to control infection across centres caring for patients with PCD. Within the BEAT-PCD network, COST Action and ERS CRC together with the ERN-Lung PCD core a first initiative has now been taken towards creating such a consensus statement. Methods A multidisciplinary international PCD expert panel was set up to create a consensus statement for infection prevention and control (IP&C) for PCD, covering …

:Otros calificadores::Otros calificadores::/prevención & control [Otros calificadores]Infeccions respiratòries en els infantsPrimary Ciliary DyskinesiaStatement (logic)Respiratory SystemMULTICENTERRECOMMENDATIONS0302 clinical medicineOriginal Research ArticlesPandemicSTENOTROPHOMONAS-MALTOPHILIAInfection control:Pathological Conditions Signs and Symptoms::Pathologic Processes::Disease Attributes::Chronic Disease [DISEASES]030212 general & internal medicinePrimary ciliary dyskinesiaMalalties transmissibles - Transmissió:Environmental Health::Health::Environmental Illness::Communicable Diseases::Communicable Disease Control [PUBLIC HEALTH]FOUNDATIONbiologyTransmission (medicine)R:Congenital Hereditary and Neonatal Diseases and Abnormalities::Congenital Abnormalities::Abnormalities Multiple::Ciliopathies::Congenital Hereditary and Neonatal Diseases and Abnormalities::Ciliary Motility Disorders [DISEASES]:afecciones patológicas signos y síntomas::procesos patológicos::atributos de la enfermedad::enfermedad crónica [ENFERMEDADES]:salud ambiental::salud::enfermedad ambiental::enfermedades transmisibles::control de enfermedades transmisibles [SALUD PÚBLICA]medicine.anatomical_structureMedicinePatient representativesLife Sciences & BiomedicinePulmonary and Respiratory Medicinemedicine.medical_specialty3610 Medicine & health:enfermedades y anomalías neonatales congénitas y hereditarias::anomalías congénitas::anomalías múltiples::ciliopatías::enfermedades y anomalías neonatales congénitas y hereditarias::trastornos de la motilidad ciliar [ENFERMEDADES]:Other subheadings::Other subheadings::/prevention & control [Other subheadings]03 medical and health sciencesotorhinolaryngologic diseasesmedicineIntensive care medicineScience & TechnologyCYSTIC-FIBROSISLungbusiness.industryPSEUDOMONAS-AERUGINOSAMalalties cròniques - PrevencióCAREEFFICACYmedicine.diseasebiology.organism_classificationrespiratory tract diseases030228 respiratory systemNontuberculous mycobacteria610 Medizin und GesundheitbusinessERJ Open Research
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Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor

2017

Prevenció; Atac d'angioedema; Inhibidor C1 Prevención; Ataque de angioedema; Inhibidor C1 Prevention; Angioedema attack; C1 inhibitor BACKGROUND: Hereditary angioedema is a disabling, potentially fatal condition caused by deficiency (type I) or dysfunction (type II) of the C1 inhibitor protein. In a phase 2 trial, the use of CSL830, a nanofiltered C1 inhibitor preparation that is suitable for subcutaneous injection, resulted in functional levels of C1 inhibitor activity that would be expected to provide effective prophylaxis of attacks. METHODS: We conducted an international, prospective, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, phase 3 trial to evaluate the …

Male:aminoácidos péptidos y proteínas::péptidos::serpinas::proteínas inactivadoras del complemento C1::proteína inhibidora del complemento C1 [COMPUESTOS QUÍMICOS Y DROGAS]0301 basic medicine:Otros calificadores::Otros calificadores::/prevención & control [Otros calificadores]Enzims proteolítics - InhibidorsSelf AdministrationSeverity of Illness Indexlaw.inventionC1-inhibitorSubcutaneous injection0302 clinical medicineRandomized controlled triallawCross-Over StudiesHereditary Angioedema Types I and IIbiologyEdema - PrevencióGeneral Medicine:Cardiovascular Diseases::Vascular Diseases::Angioedema::Angioedemas Hereditary [DISEASES]AnesthesiaHereditary angioedema:Other subheadings::Other subheadings::/administration & dosage [Other subheadings]Female:Amino Acids Peptides and Proteins::Peptides::Serpins::Complement C1 Inactivator Proteins::Complement C1 Inhibitor Protein [CHEMICALS AND DRUGS]medicine.symptomComplement C1 Inhibitor Protein:enfermedades cardiovasculares::enfermedades vasculares::angioedema::angioedemas hereditarios [ENFERMEDADES]AdultRiskmedicine.medical_specialtyInjections Subcutaneous:Aminoácidos Péptidos y Proteínas::Péptidos::Serpinas::Proteínas Inactivadoras del Complemento 1::Proteína Inhibidora del Complemento C1 [COMPUESTOS QUÍMICOS Y DROGAS]Placebo:Other subheadings::Other subheadings::/prevention & control [Other subheadings]03 medical and health sciencesDouble-Blind MethodInternal medicinemedicineHumans:Therapeutics::Drug Therapy::Drug Administration Routes::Injections::Injections Subcutaneous [ANALYTICAL DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT]:Otros calificadores::Otros calificadores::/administración & dosificación [Otros calificadores]Dose-Response Relationship DrugAngioedemabusiness.industry:Terapéutica::Tratamiento Farmacológico::Vías de Administración de Medicamentos::Inyecciones::Inyecciones Subcutáneas [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS]Injeccions hipodèrmiquesmedicine.diseaseCrossover studyClinical trial:terapéutica::farmacoterapia::vías de administración de medicamentos::inyecciones::inyecciones subcutáneas [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS]030104 developmental biology030228 respiratory systembiology.proteinbusinessNew England Journal of Medicine
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The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN

2022

Primary ciliary dyskinesia; Rare genetic disorder; Lung diseases Discinesia ciliar primaria; Trastorno genético raro; Enfermedades pulmonares Discinesia ciliar primària; Trastorn genètic rar; Malalties pulmonars Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by impaired mucociliary clearance leading to irreversible lung damage. In contrast to other rare lung diseases like cystic fibrosis (CF), there are only few clinical trials and limited evidence-based treatments. Management is mainly based on expert opinions and treatment is challenging due to a wide range of clinical manifestations and disease severity. To improve clinical and translational research and facili…

Pulmonary and Respiratory Medicine:enfermedades respiratorias::trastornos de la motilidad ciliar [ENFERMEDADES]:Health Occupations::Medicine::Pediatrics [DISCIPLINES AND OCCUPATIONS]Respiratory SystemSağlık BilimleriClinical Medicine (MED)SOLUNUM SİSTEMİRespiratory CareHealth SciencesMANAGEMENTKlinik Tıp (MED)Chest Diseases and Allergy:profesiones sanitarias::medicina::pediatría [DISCIPLINAS Y OCUPACIONES]:Otros calificadores::/terapia [Otros calificadores]:Respiratory Tract Diseases::Ciliary Motility Disorders [DISEASES]Internal Medicine SciencesScience & TechnologyKlinik TıpMUTATIONSRESPIRATORY SYSTEM:Other subheadings::/therapy [Other subheadings]Dahili Tıp BilimleriGöğüs Hastalıkları ve AllerjiCLINICAL MEDICINECèl·lules - Motilitat:Congenital Hereditary and Neonatal Diseases and Abnormalities::Genetic Diseases Inborn [DISEASES]TıpPulmons - Malalties - TractamentAkciğer ve Solunum TıbbıMedicineMalalties congènites:enfermedades y anomalías neonatales congénitas y hereditarias::enfermedades genéticas congénitas [ENFERMEDADES]Solunum BakımıLife Sciences & BiomedicinePulmons Malalties
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