von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in VWD patients treated with Voncento in the United Kingdom (UK). A Markov structure was developed to estimate quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon.
View Article and Find Full Text PDFBackground: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection.
Objectives: Determine valoctocogene roxaparvovec durability, efficacy, and safety 4 years after treatment.
Methods: In the phase 3 GENEr8-1 trial, 134 adult male persons with severe hemophilia A without inhibitors and previously using FVIII prophylaxis received a 6 × 10 vg/kg infusion of valoctocogene roxaparvovec.
Background: Recruiting older adults into clinical trials can be particularly challenging. Our objective was to determine if targeted web-based advertising is an effective recruitment strategy.
Methods: We compared the recruitment rates of traditional and targeted web-based methods for three representative clinical trials involving older adults.
Elevation of intraocular pressure (IOP) due to trabecular meshwork (TM) dysfunction, leading to neurodegeneration, is the pathological hallmark of primary open-angle glaucoma (POAG). Impaired axonal transport is an early and critical feature of glaucomatous neurodegeneration. However, a robust mouse model that replicates these human POAG features accurately has been lacking.
View Article and Find Full Text PDFPurpose: Addressing the retention challenges faced by non-metropolitan speech-language pathologists (SLPs) is crucial for achieving health services equity in Australia. This study assesses the applicability of previous literature findings to the current context of Australian SLPs. Furthermore, identifying specific factors influencing the retention of SLPs in outer regional, remote, and very remote areas of Australia.
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