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Blocking Notch3 Signaling Abolishes MUC5AC Production in Airway Epithelial Cells from AsthmaticsWe demonstrate that NOTCH3 is a regulator of MUC5AC production
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Longitudinal trajectories of BMI z-score: an international comparison of 11,513 Australian, American and German/Austrian/Luxembourgian youth with type 1 diabetesThis multinational study presents unique body mass index z score trajectories in youth with type 1 diabetes across three continents
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Medication adherence during adjunct therapy with statins and ACE inhibitors in adolescents with type 1 diabetesWe report an overall good adherence to ACE inhibitors and statins during a clinical trial, although there was a clear decline in adherence over time
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SCN1A Variants in vaccine-related febrile seizures: A prospective studyPathogenic SCN1A variants may be identified in infants with vaccine-proximate febrile seizures
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Association of suicidal behavior with exposure to suicide and suicide attempt: A systematic review and multilevel meta-analysisprior exposure to suicide and prior exposure to suicide attempt in the general population are associated with increased odds of subsequent suicidal behavior
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The Undiagnosed Diseases Network International: Five years and moreThe Undiagnosed Diseases Network International was established with the major aims of providing diagnoses to patients
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Understanding group A streptococcal pharyngitis and skin infections as causes of rheumatic fever: Protocol for a prospective disease incidence studyThis study will identify the incidence of true Group A Streptococcal pharyngitis and serological responses to Group A Streptococcal GAS skin infections
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RD-RAP: Beyond rare disease patient registries, devising a comprehensive data and analytic frameworkWe introduce and describe the concept of a Rare Disease Registry and Analytics Platform
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Challenges and considerations for antifungal prophylaxis in children with acute myeloid leukemiaChildren receiving treatment for acute myeloid leukemia (AML) are at high risk of invasive fungal disease (IFD). Evidence from pediatric studies support the efficacy of antifungal prophylaxis in reducing the burden of IFD in children receiving therapy for AML, yet existing antifungal agents have specific limitations and comparative data to inform the optimal prophylactic approach are lacking.
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Autism likelihood in infants born to mothers with asthma is associated with blood inflammatory gene biomarkers in pregnancyMothers with asthma or atopy have a higher likelihood of having autistic children, with maternal immune activation in pregnancy implicated as a mechanism. This study aimed to determine, in a prospective cohort of mothers with asthma and their infants, whether inflammatory gene expression in pregnancy is associated with likelihood of future autism.