Current medical research and opinion
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The objective was to investigate the severity of aromatic L-amino acid decarboxylase deficiency (AADCd) as reported in the published literature and to collate evidence of the clinical manifestations of AADCd, and the impact of the disease on patients, caregivers, and healthcare systems. ⋯ Irrespective of the ambulatory status of individuals, AADCd is a debilitating disease that significantly impacts quality-of-life for individuals and caregivers. It impacts the healthcare system due to the need for complex coordinated activities of a multidisciplinary specialist team.
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Review Meta Analysis
Relative efficacy of varicella vaccines: Network meta-analysis of randomized controlled trials.
Although varicella vaccination is highly effective, no head-to-head randomized controlled trials (RCTs) have compared the efficacy of different vaccine formulations. This study assessed the relative efficacy of different varicella vaccines using network meta-analysis (NMA). ⋯ Our study reported higher VE for two-dose V-MSD and MMRV-GSK. Among one-dose formulations, one-dose V-MSD was more efficacious than one-dose V-GSK. Policymakers should take into consideration differences in VE when implementing one- versus two-dose strategies in universal vaccination programs.
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The purpose of this study was to compare the effectiveness of the only Food and Drug Administration-authorized prescription digital therapeutic (PDT) Somryst versus face-to-face cognitive behavioral therapy for insomnia (CBT-I), or FDA-approved prescription medications for insomnia. ⋯ This network meta-analysis demonstrated that a PDT delivering CBT-I had the highest probability of being most effective compared to face-to-face CBT-I, prescription sleep medications, or placebo, as measured by reductions in mean ISI score from baseline and ISI-determined remittance.
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Friedreich ataxia (FA) is a rare, inherited neuromuscular disease characterized by an early onset and progressive limb and gait ataxia. Currently, there are no approved treatments for FA. It is important to understand the burden of FA, including its extent and the most salient elements. The objective of this study is therefore to systematically review the literature regarding the aspects of prevalence, health-related quality of life (HRQoL), and economic outcomes that are associated with FA, and to subsequently identify relevant knowledge gaps. ⋯ Findings from this systematic review revealed several knowledge gaps that would preclude the conduct of a robust assessment of the benefits and outcomes associated with a disease-modifying FA therapy. Additional understanding regarding patient and caregiver HRQoL and costs is required.
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To examine whether QT interval shortening is an overlooked adverse event as compared to QT prolongation through a review of preclinical, clinical and post-marketing adverse event data available to the regulator for centrally and nationally authorized medicinal products. ⋯ From a regulatory perspective one cannot refute the possibility of a clinically relevant risk from QT shortening through the current testing requirements. Lack of further investigations into any potential morphological changes to the AP, or APD90 shortening beyond a specified threshold in our opinion does not fully exclude the possibility of proarrhythmic effects of active substances.