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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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Cause-Specific Secular Trends and Prevention Measures of Post-Neonatally Acquired Cerebral Palsy in Victoria and Western Australia 1975–2014: A Population-Based Observational StudyTo describe the timing and causes of post-neonatally acquired cerebral palsy (PNN-CP) and map the implementation of relevant preventive strategies against cause-specific temporal trends in prevalence.
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Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trialCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes.
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Research enriching the lives of girls with Rett syndromeA program developed by The Kids Research Institute Australia researcher Dr Jenny Downs has led to dramatic improvements in the functioning of Chinese children with Rett syndrome, and could change the world.
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A Population-Based Matched-Sibling Analysis Estimating the Associations Between First Interpregnancy Interval and Birth OutcomesInterpregnancy intervals of <6 months were associated with increased odds of preterm birth in second-born infants
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Management of oral and dental problems in Rett syndrome: a narrative review of the literatureReview of the available dental literature on assessment and management of the oral manifestations of Rett syndrome
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We examined the impact of introducing a dedicated team to OPAT, to define the role of increased medical oversight in improving patient outcomes in this cohort.To evaluate the risk of stillbirth, PTB, and SGA as a proxy for FGR following exposure to one or more of these factors in a previous birth.
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Cannabis for refractory epilepsy in children: A review focusing on CDKL5 Deficiency DisorderThis review provides the first comprehensive overview of the potential role for cannabis based preparations in the treatment of CDKL5 Deficiency Disorder
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Longitudinal effects of caregiving on parental well-being: the example of Rett syndrome, a severe neurological disorderOur findings suggest that some opportunities do exist for clinicians to help optimise parental well-being
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Risk factors for hospitalizations associated with depression among women duringthe years around a birth: a retrospective cohort studyResults provide preliminary evidence that barriers to treating depression with anti-depressants in mothers from low income areas during the years around a birth