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Combining app-based behavioral therapy with electronic cigarettes for smoking cessation: a study protocol for a single-arm mixed-methods pilot trial
BackgroundCigarette smoking remains a leading cause of preventable illness and death, underscoring the need for effective evidence-based smoking...
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A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem
People with rare lysosomal storage diseases face challenges in their care that arise from disease complexity and heterogeneity, compounded by many...
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The STAR Compass to Guide Future Pharmacovigilance Based on a 10-Year Review of the Strengthened EU System
This article reflects on the 2010 pharmacovigilance legislation of the European Union (EU). Its legislative aim of better patient and public health...
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Regorafenib plus FOLFIRINOX as first-line treatment for patients with RAS-mutant metastatic colorectal cancer (FOLFIRINOX-R trial): a dose-escalation study
PurposeThe combination of bevacizumab and FOLFIRINOX is used in patients with RAS-mutant metastatic colorectal cancer (RASm-mCRC). Regorafenib, an...
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The Association of Gabapentin Initiation with Cognitive and Behavioral Changes in Older Adults with Cognitive Impairment: A Retrospective Cohort Study
BackgroundAlthough gabapentin has been increasingly prescribed to older adults, the relation between gabapentin initiation and longer-term...
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Unveiling the untreated: development of a database algorithm to identify potential Fabry disease patients in Germany
BackgroundFabry disease (FD), an X-linked lysosomal storage disorder, is caused by mutations in the gene encoding α-galactosidase A, resulting in...
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Essential components of an effective transition from paediatric to adult neurologist care for adolescents with Duchenne muscular dystrophy; a consensus derived using the Delphi methodology in Eastern Europe, Greece and Israel
PurposeAn increasing number of patients with Duchenne muscular dystrophy (DMD) now have access to improved standard of care and disease modifying...
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Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study
BackgroundHypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade ® , an...
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Lichen Planus: What is New in Diagnosis and Treatment?
Lichen planus (LP), an idiopathic, multifaceted chronic inflammatory disease with a heterogeneous clinical presentation, affects approximately 0.5–1%...
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Pre-clinical Models for Geriatric Pharmacotherapy
With ageing of the population worldwide and discovery of new medications for prevention and management of age-related conditions, there is increasing...
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Efficacy and safety of D-penicillamine, trientine, and zinc in pediatric Wilson disease patients
ObjectivesWilson disease (WD) is a rare genetic disease affecting copper metabolism and the biliary tract’s copper excretion. Lifelong medication is...
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Use of Muscle Relaxants After Surgery in Traditional Medicare Part D Enrollees
BackgroundSurgeons have come under increased scrutiny for postoperative pain management, particularly for opioid prescribing. To decrease opioid use...
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Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series
BackgroundHereditary angioedema (HAE) is a rare and potentially life-threatening disease that manifests clinically as recurrent episodes of swelling...
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A Calculated Risk: Evaluation of QTc Drug–Drug Interaction (DDI) Clinical Decision Support (CDS) Alerts and Performance of the Tisdale Risk Score Calculator
IntroductionA risk factor for a potentially fatal ventricular arrhythmia Torsade de Pointes is a prolongation in the heart rate-corrected QT interval...
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Optimal Significance Levels and Sample Sizes for Signal Detection Methods Based on Non-constant Hazards
Background and ObjectivesStatistical methods for signal detection of adverse drug reactions (ADRs) in electronic health records (EHRs) need...
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Impacts of X-linked Retinitis Pigmentosa and Patient Pathways in European Countries: Results from the Cross-sectional EXPLORE XLRP-1 Physician Survey
IntroductionX-linked retinitis pigmentosa (XLRP) is a rare, incurable, vision-threatening, genetic disease. In this study, we aimed to reveal the...
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Increased Risk of Severe Cardiovascular Events Following Exacerbations of Chronic Obstructive Pulmonary Disease: Results of the EXACOS-CV Study in Japan
IntroductionSevere exacerbations of chronic obstructive pulmonary disease (COPD) are known to increase the risk of cardiovascular events. However,...
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Usability of the SB11 Pre-filled Syringe (PFS) in Patients with Retinal Diseases
IntroductionSB11 (Byooviz™; Samsung Bioepis Co., Ltd.) is a ranibizumab (Lucentis ® ; Genentech, Inc.) biosimilar targeting vascular endothelial...
