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Adapting a measure of gross motor skills for individuals with CDKL5 deficiency disorder: A psychometric studyValidated measures capable of demonstrating meaningful interventional change in the CDKL5 deficiency disorder (CDD) are lacking. The study objective was to modify the Rett Syndrome Gross Motor Scale (RSGMS) and evaluate its psychometric properties for individuals with CDD.
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Short Message Service Reminder Nudge for Parents and Influenza Vaccination Uptake in Children and Adolescents with Special Risk Medical Conditions: The Flutext-4U Randomized Clinical TrialChildren with chronic medical conditions are at increased risk of severe influenza. Uptake of influenza vaccination in children and adolescents with these identified special risk medical conditions is suboptimal.
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No association between in utero exposure to emissions from a coalmine fire and post-natal lung functionStudies linking early life exposure to air pollution and subsequent impaired lung health have focused on chronic, low-level exposures in urban settings. We aimed to determine whether in utero exposure to an acute, high-intensity air pollution episode impaired lung function 7-years later.
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Blinatumomab Added to Chemotherapy in Infant Lymphoblastic LeukemiaKMT2A-rearranged acute lymphoblastic leukemia (ALL) in infants is an aggressive disease with 3-year event-free survival below 40%. Most relapses occur during treatment, with two thirds occurring within 1 year and 90% within 2 years after diagnosis. Outcomes have not improved in recent decades despite intensification of chemotherapy.
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The effect of CFTR modulators on structural lung disease in cystic fibrosisNewly developed quantitative chest computed tomography (CT) outcomes designed specifically to assess structural abnormalities related to cystic fibrosis (CF) lung disease are now available. CFTR modulators potentially can reduce some structural lung abnormalities. We aimed to investigate the effect of CFTR modulators on structural lung disease progression using different quantitative CT analysis methods specific for people with CF (PwCF).
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Bivalent Prefusion F Vaccine in Pregnancy to Prevent RSV Illness in InfantsWhether vaccination during pregnancy could reduce the burden of respiratory syncytial virus (RSV)-associated lower respiratory tract illness in newborns and infants is uncertain.
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Spatiotemporal distributions of under-five mortality in Ethiopia between 2000 and 2019Under-five mortality declined in the last two decades in Ethiopia, but sub-national and local progress remains unclear. This study aimed to investigate the spatiotemporal distributions and ecological level factors of under-five mortality in Ethiopia. Data on under-five mortality were obtained from five different Ethiopian Demographic and Health Surveys conducted in 2000, 2005, 2011, 2016, and 2019. Environmental and healthcare access data were obtained from different publicly available sources.
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Impact of COVID-19 on mental health in Bhutan: a way forward for actionThe coronavirus disease 2019 (COVID-19) pandemic continues to impact mental health and wellbeing globally. There is a lack of scientific documentation highlighting the mental health impact of COVID-19 in Bhutan. We present the mental health burdens and control measures taken, and suggest ways to further strengthen mental health services in Bhutan.
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Evaluating the role of asymptomatic throat carriage of Streptococcus pyogenes in impetigo transmission in remote Aboriginal communities in Northern Territory, Australia: a retrospective genomic analysisStreptococcus pyogenes, or group A Streptococcus (GAS), infections contribute to a high burden of disease in Aboriginal Australians, causing skin infections and immune sequelae such as rheumatic heart disease. Controlling skin infections in these populations has proven difficult, with transmission dynamics being poorly understood. We aimed to identify the relative contributions of impetigo and asymptomatic throat carriage to GAS transmission.
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In vivo loss of tumorigenicity in a patient-derived orthotopic xenograft mouse model of ependymomaEpendymomas (EPN) are the third most common malignant brain cancer in children. Treatment strategies for pediatric EPN have remained unchanged over recent decades, with 10-year survival rates stagnating at just 67% for children aged 0-14 years. Moreover, a proportion of patients who survive treatment often suffer long-term neurological side effects as a result of therapy. It is evident that there is a need for safer, more effective treatments for pediatric EPN patients.