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Palovarotene in fibrodysplasia ossificans progressiva: review and perspective.
Expert Opin Pharmacother
January 2025
Amsterdam University Medical Center (Amsterdam UMC), location VU University Medical Center (VUMC), Amsterdam, Netherlands.
Introduction: Palovarotene is a retinoic acid receptor gamma agonist that was studied in phase-2 and phase-3 clinical trials for the inhibition of new heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP). Despite numerous setbacks and regulatory delays, palovarotene is now the first approved FOP treatment in the U.S.
View Article and Find Full Text PDFPatient-driven personal importation in the face of cost barriers to care: can we do better?
Intern Med J
January 2025
Faculty of Medicine, UNSW Sydney, St Vincent's Clinical School, Sydney, New South Wales, Australia.
Australia has a robust public health system that helps to make medicines affordable. However, evidence shows that a significant proportion of Australians still cannot afford medicines prescribed to them and that some patients import medicines from abroad as a result. The strongest predictor of whether patients import medicines is whether they have discussed it with their doctor.
View Article and Find Full Text PDFComparison of Oncology Drug Lag in Japan and South Korea Based on the Interval between the U.S. Approval and the Local Approval.
Biol Pharm Bull
January 2025
Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo 108-8641, Japan.
Drug lag is a serious issue for patients with life-threatening diseases such as cancer. Japan and Korea have been facing a large drug lag, despite having a large market and a good clinical trial environment. We analyzed drug lags for anticancer drugs between these countries, using the information on 82 anticancer drugs approved in the United States between 2017 and 2022.
View Article and Find Full Text PDFEfficacy and safety of disease-modifying therapies in pediatric-onset multiple sclerosis: A systematic review of clinical trials and observational studies.
Mult Scler Relat Disord
January 2025
Department of Nutrition and Drug Research, Faculty of Health Sciences, Institute of Public Health, Jagiellonian University Medical College, Skawińska Street 8, 31-066 Krakow, Poland. Electronic address:
Objective: This study aimed to review the efficacy and safety profile of disease-modifying therapies (DMTs) in patients with relapsing pediatric-onset multiple sclerosis (POMS).
Methods: A systematic review was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Published randomized controlled trials (RCTs), nonrandomized studies with a control group, large single-arm studies, and ongoing (unpublished) studies investigating the use of approved and unapproved DMTs in POMS were included.
Compounded glucagon-like peptide-1 receptor agonists for weight loss: the direct-to-consumer market in Colorado.
J Pharm Policy Pract
December 2024
Department of Clinical Pharmacy, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Background: High prices and other access barriers have contributed to the rise of a market for compounded glucagon-like peptide-1 receptor agonists for weight loss in the United States. This market has not been systematically studied. We conducted a pilot study to assess the prevalence, characteristics, and advertising content of direct-to-consumer providers of compounded glucagon-like peptide-1 products for weight loss in Colorado.
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