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Development of an International Database for a Rare Genetic Disorder: The MECP2 Duplication Database (MDBase)The natural history of MECP2 duplication syndrome (MDS), a rare X-linked neurodevelopmental disorder with an estimated birth prevalence of 1/150,000 live births, is poorly understood due to a lack of clinical data collected for research. Such information is critical to the understanding of disease progression, therapeutic endpoints and outcome measures for clinical trials, as well as the development of therapies and orphan products.
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Revaccination with Bacille Calmette-Guérin (BCG) is associated with an increased risk of abscess and lymphadenopathyThe reported frequency and types of adverse events following initial vaccination and revaccination with Bacille Calmette-Guérin (BCG) varies worldwide. Using active surveillance in a randomised controlled trial of BCG vaccination (the BRACE trial), we determined the incidence and risk factors for the development of BCG injection site abscess and regional lymphadenopathy.
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Lactoferrin Expression Is Not Associated with Late-Onset Sepsis in Very Preterm InfantsPreterm infants are at a high risk of developing late-onset sepsis (LOS). Lactoferrin is one of the most abundant endogenous antimicrobial proteins expressed in breast milk, stools, and blood, and a candidate for preventive intervention. Large clinical trials have recently investigated whether enteral supplementation with bovine lactoferrin reduces LOS.
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Safety and immunogenicity of a primary series and booster dose of the meningococcal serogroup B-factor H binding protein vaccine (MenB-FHbp) in healthy children aged 1–9 years: two phase 2 randomised, controlled, observer-blinded studiesThe meningococcal serogroup B-factor H binding protein vaccine (MenB-FHbp) is licensed for use in children aged 10 years or older for protection against invasive serogroup B meningococcal disease. Because young children are at increased risk of invasive meningococcal disease, MenB-FHbp clinical data in this population are needed.
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Safety and Immunogenicity of MF59-Adjuvanted Cell Culture-Derived A/H5N1 Subunit Influenza Virus Vaccine: Dose-Finding Clinical Trials in Adults and the ElderlyIn adult and elderly participants, the full-dose aH5N1c vaccine formulation was well tolerated and met US and European licensure criteria for pandemic vaccines
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Geographical disparities in emergency department presentations for acute respiratory infections and risk factors for presentingOne in four ED presentations in WA children are for ARIs, representing a significant out-of-hospital burden with some evidence of geographical disparity
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Duplex quantitative PCR assay for detection of haemophilus influenzae that distinguishes fucose-and protein d-negative strainsDeveloped a specific Haemophilus influenzae quantitative PCR (qPCR) that also identifies fucose-negative and protein D-negative strains
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Immunogenicity and clinical effectiveness of the trivalent inactivated influenza vaccine in immunocompromised children undergoing treatment for cancerThe trivalent inactivated influenza vaccine is safe, immunogenic, provides clinical protection and should be administered annually to immunosuppressed children receiving treatment for cancer
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Bacillus Calmette-Guérin vaccination for protection against recurrent herpes labialis: a nested randomised controlled trialRecurrences of herpes simplex virus (HSV) in the orofacial region (herpes labialis or cold sores) impact quality-of-life. We aimed to study whether the bacille Calmette-Guérin (BCG) vaccine can attenuate cold sore recurrences through off-target immunomodulatory effects.
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FeBRILe3: Risk-Stratification and Diagnosis of Serious Bacterial Infections in Febrile Infants Less Than 3 Months OldEvidence-based recommendations exist for early discharge (before 48 h) of young infants with fever without source (FWS) at low risk of serious bacterial infections (SBIs). However, concerns regarding the applicability of international data to local contexts may hinder implementation. We aimed to describe the local epidemiology of FWS and evaluate a newly implemented risk-stratification guideline to support practice change.