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An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database.
Genes (Basel)
November 2024
Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
Background/objectives: Antisense oligonucleotide (ASO)-mediated exon-skipping is an effective approach to restore the disrupted reading frame of the dystrophin gene for the treatment of Duchenne muscular dystrophy (DMD). Currently, four FDA-approved ASOs can target three different exons, but these therapies are mutation-specific and only benefit a subset of patients. Understanding the broad applicability of exon-skipping approaches is essential for prioritizing the development of additional therapies with the greatest potential impact on the DMD population.
View Article and Find Full Text PDFPreferences for speed of access versus certainty of the survival benefit of new cancer drugs: a discrete choice experiment.
Lancet Oncol
December 2024
Department of Health Policy, London School of Economics and Political Science, London, UK.
Background: The extent to which patients with cancer are willing to accept uncertainty about the clinical benefit of new cancer drugs in exchange for faster access is not known. This study aims to examine preferences for access versus certainty, and to understand factors that influence these preferences.
Methods: A US nationally representative sample of older adults were recruited via Cint, an online platform for survey research, to take part in an online discrete choice experiment.
Impact of age, race, and medication use on efficacy endpoints in a randomized controlled trial of topical sildenafil cream for the treatment of female sexual arousal disorder.
Sex Med
October 2024
Daré Bioscience, San Diego, CA, United States.
Background: A study of topical Sildenafil Cream 3.6% was completed among healthy premenopausal women with female sexual arousal disorder.
Aims: To compare efficacy endpoints based on product use in pre-planned and post-hoc subsets of age, race, and medication use.
HPV16/18 antibodies 16-years after single dose of bivalent HPV vaccination: Costa Rica HPV vaccine trial.
J Natl Cancer Inst Monogr
November 2024
Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD, USA.
Background: The Costa Rica HPV Vaccine Trial provided initial evidence that 1 dose of the bivalent human papillomavirus (HPV) vaccine induces stabilizing antibody levels that may provide extended protection against HPV-16/18 infections. We report antibody seropositivity and stability 11 to 16 years after vaccination.
Methods: We invited a random subset of Costa Rica HPV Vaccine Trial participants (n = 398) who had received 3 doses and all women (n = 203) who had received 1 dose at 18 to 25 years of age to follow-up visits 11, 14, and 16 years after vaccination.
Objective: E-cigarette use is associated with subsequent cigarette smoking among youth. The current study examined the mediating role of social-cognitive factors in this association.
Methods: Data from four waves of the Population Assessment of Tobacco and Health study (2013/4 - 2017/8) were analyzed.
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