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Research
International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USAThe clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.
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Developmental Mismatch Across Brain Modalities in Young ChildrenBrain development during the preschool period is complex and extensive and underlies ongoing behavioral and cognitive maturation. Increasing understanding of typical brain maturation during this time is critical to early identification of atypical development and could inform treatments and interventions.
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Streptococcus pyogenes pharyngitis elicits diverse antibody responses to key vaccine antigens influenced by the imprint of past infectionsKnowledge gaps regarding human immunity to Streptococcus pyogenes have impeded vaccine development. To address these gaps and evaluate vaccine candidates, we established a human challenge model of S. pyogenes pharyngitis. Here, we analyse antibody responses in serum and saliva against 19 antigens to identify characteristics distinguishing 19 participants who developed pharyngitis and 6 who did not.
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A pilot evaluation of school-based LEGO robotics therapy for autistic studentsThere is emerging evidence that LEGO® therapy is an effective way of supporting younger autistic children develop their communication and social skills. LEGO® robotics therapy - which uses the principles of LEGO® therapy applied to LEGO® robotics - may be an age-appropriate intervention to reduce anxiety and increase social skills in autistic adolescents.
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A matter of timing: Biting by malaria-infected Anopheles mosquitoes and the use of interventions during the night in rural southeastern TanzaniaKnowing when and where infected mosquitoes bite is required for estimating accurate measures of malaria risk, assessing outdoor exposure, and designing intervention strategies. This study combines secondary analyses of a human behaviour survey and an entomological survey carried out in the same area to estimate human exposure to malaria-infected Anopheles mosquitoes throughout the night in rural villages in south-eastern Tanzania.
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Ion channel modulator DPI-201-106 significantly enhances antitumor activity of DNA damage response inhibitors in glioblastomaGlioblastoma, a lethal high-grade glioma, has not seen improvements in clinical outcomes in nearly 30 years. Ion channels are increasingly associated with tumorigenesis, and there are hundreds of brain-penetrant drugs that inhibit ion channels, representing an untapped therapeutic resource. The aim of this exploratory drug study was to screen an ion channel drug library against patient-derived glioblastoma cells to identify new treatments for brain cancer.
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Impact of integrating traditional care with the modern healthcare system in reducing tuberculosis diagnosis delays in Ethiopia: a clustered randomized controlled studyDiagnosis and treatment initiation delays for tuberculosis (TB) are significant challenges in resource-limited settings. These delays can result in poor treatment outcomes, disease transmission, and increased costs. This study aimed to assess the effect of integrating traditional care with modern healthcare systems on reducing TB diagnosis delay.
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Effects of dog ownership on children’s social-emotional development: findings from the PLAYCE cohort studyDog ownership is common in families with children and could play a role in children's social-emotional development. This study used longitudinal data on dog ownership and changing dog ownership to investigate their effects on young children's social-emotional development.
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Pattern of hospital admissions and costs associated with acute rheumatic fever and rheumatic heart disease in Australia, 2012–2017This study aims to describe the pattern and trends in acute rheumatic fever (ARF)/rheumatic heart disease (RHD)-related hospitalisations and costs for Australians aged <65 years.
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Systematic Review of Household Transmission of Strep A: A Potential Site for Prevention That Has Eluded AttentionAlthough Streptococcus pyogenes (Strep A) is the sixth-most common infectious disease globally, its transmission within the household remains an understudied driver of infection. We undertook a systematic review to better understand the transmission of Strep A among people within the home, while highlighting opportunities for prevention.