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National survey of financial burden and experience among patients with cancer and autoimmune disease receiving charitable copay assistance.
Cancer
January 2025
Fox Chase Cancer Center, Temple University Health System, Philadelphia, Pennsylvania, USA.
Background: Little is known about the role that charitable copay assistance (CPA) plays in addressing access to care and financial distress. The study sought to evaluate financial distress and experience with CPA among patients with cancer and autoimmune disease.
Methods: This is a national cross-sectional self-administered anonymous electronic survey conducted among recipients of CPA to cover the costs of a drug for cancer or autoimmune disease.
Prevalence, Patient Characteristics, and Treatment of Patients with Hypertrophic Cardiomyopathy: A Nationwide Payer Database Study.
Cardiol Ther
January 2025
Bristol Myers Squibb, Tokyo, Japan.
Introduction: Data on the prevalence of hypertrophic cardiomyopathy (HCM), characteristics of patients with HCM, and treatment patterns in Japan are limited. This study aimed to estimate the prevalence of HCM and describe the patient characteristics, treatment patterns, and utilization of medical expense subsidies in Japan, using payer claims data from insurers.
Methods: This retrospective study of patients with HCM in Japan utilized payer claims data from insurers (Advanced Elderly Medical Service System [AEMSS], Kokuho, and Kempo) from January 1, 2017, to December 31, 2021.
Longitudinal qualitative assessment of meaningful symptoms and relevance of WATCH-PD digital measures for people with early Parkinson's.
J Neurol
January 2025
Center for Health + Technology, University of Rochester Medical Center, Rochester, NY, USA.
Background: Longitudinal qualitative data on what matters to people with Parkinson's disease are lacking and needed to guide patient-centered clinical care and development of outcome measures.
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Improving large language model applications in biomedicine with retrieval-augmented generation: a systematic review, meta-analysis, and clinical development guidelines.
J Am Med Inform Assoc
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Department of Biomedical Informatics, Vanderbilt University Medical Center, Nashville, TN 37212, United States.
Objective: The objectives of this study are to synthesize findings from recent research of retrieval-augmented generation (RAG) and large language models (LLMs) in biomedicine and provide clinical development guidelines to improve effectiveness.
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Cenobamate as add-on treatment for SCN8A developmental and epileptic encephalopathy.
Epilepsia
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Department of Epilepsy Genetics and Personalized Medicine, Danish Epilepsy Center, Dianalund, Denmark.
Objectives: Developmental and epileptic encephalopathies (DEEs) caused by pathogenic variants in SCN8A are associated with difficult-to-treat and early-onset seizures, developmental delay/intellectual disability, impaired quality of life, and increased risk of early mortality. High doses of sodium channel blockers are typically used to treat SCN8A-DEE caused by gain-of-function (GoF) variants. However, seizures are often drug resistant, and only a few patients achieve seizure freedom.
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