See related editorial by Lundebjerg et al.
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http://dx.doi.org/10.1111/jgs.17530 | DOI Listing |
Introduction: Vosoritide is the first approved treatment for achondroplasia, a rare genetic disorder that results in disproportionate short stature. In clinical trials, vosoritide has shown a positive safety profile and increased height in children with achondroplasia. This paper shares the organizational structure, initiation, follow-up protocol, and findings of a vosoritide early access program (EAP) conducted in France.
View Article and Find Full Text PDFPublic Health
January 2025
Health Research Institute, Faculty of Health, University of Canberra, ACT, Australia.
Objectives: Health and social service research impact analysis play a pivotal role in demonstrating research value. Impact analysis of programs, interventions, or policies in real-world settings is complex. There are many implementation evaluation theories, models, and frameworks (TMF) and researchers find choosing one challenging.
View Article and Find Full Text PDFEur J Clin Microbiol Infect Dis
January 2025
Laboratory of Medical Microbiology, Ghent University Hospital, Ghent, Belgium.
Purpose: Mortality and morbidity of patients with bloodstream infection (BSI) remain high despite advances in diagnostic methods and efforts to speed up reporting. This study investigated the impact of reporting rapid Minimum Inhibitory Concentration (MIC)-results in Gram negative BSIs with the ASTar system (Q-linea, Uppsala, Sweden) on the adaptation of empirically started antimicrobial therapy. We performed a real-world study during which antimicrobial susceptibility testing (AST) results were instantly reported to the treating physician in an established multidisciplinary antimicrobial stewardship setting.
View Article and Find Full Text PDFCancer Med
February 2025
Pulmonology and Thoracic Oncology Department, APHP Hôpital Tenon and Sorbonne Université, Paris, France.
Background: Real-world data regarding patients with non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion (ex20ins) mutations receiving mobocertinib are limited. This study describes these patients' characteristics and outcomes.
Methods: A chart review was conducted across three countries (Canada, France, and Hong Kong), abstracting data from eligible patients (NCT05207423).
J Med Internet Res
January 2025
Department for Prevention and Care of Diabetes, Faculty of Medicine Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Background: Digital technologies for type 2 diabetes mellitus (T2DM) care hold great potential to improve patients' health in the long term. Only a subset of telemedicine offerings are digital interventions that meet the criteria for prescribable digitale Gesundheitsanwendung (digital health apps; DiGAs) in Germany. Digital treatments further provide vast amounts of patient data that are important to generate evidence.
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