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Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients with Duchenne Muscular Dystrophy: A MarketScan Claims Analysis.
Adv Ther
November 2024
Sarepta Therapeutics, Inc, Cambridge, MA, 02142, USA.
Article Synopsis
- Phosphorodiamidate morpholino oligomers (PMOs) are weekly intravenous treatments approved for Duchenne muscular dystrophy (DMD) that allow for certain exon skipping, but real-world usage data is scarce.
- The study used data from MarketScan commercial and Medicaid claims between 2018-2021 to analyze PMO treatment patterns, finding 133 patients with claims for PMOs, generally aged around 14 years and predominantly male.
- Results showed a high median proportion of days covered at 83.4%, with over half of the patients maintaining continuous treatment coverage, and a significant majority of those with treatment gaps later resumed PMO claims despite potential underestimations from the claims data.*
Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy.
Drug Metab Dispos
November 2024
Sarepta Therapeutics, Inc., Cambridge, Massachusetts.
Article Synopsis
- Eteplirsen, golodirsen, and casimersen are drugs approved for treating Duchenne muscular dystrophy (DMD) that target specific genetic mutations associated with the disease through a mechanism called exon skipping.
- In studies, these drugs showed similar metabolism and pharmacokinetic properties across various animal models, with consistent plasma exposure and low plasma protein binding.
- The research suggests that these PMOs share key characteristics that could support the development of a broader PMO drug class, potentially leading to new treatments for genetic conditions like DMD.
Mechanisms of Action of the US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs.
BioDrugs
July 2024
Department of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, USA.
Article Synopsis
- - Antisense oligonucleotides (ASOs) are single-stranded nucleic acids that specifically target RNA and have been approved by the FDA for various diseases, utilizing three main mechanisms of action: mRNA degradation, exon skipping, and mRNA function inhibition.
- - ASOs are structurally modified for stability and resistance to degradation, and examples include drugs like inotersen for hereditary transthyretin amyloidosis and nusinersen for spinal muscular atrophy.
- - The design of ASOs is largely based on mRNA sequence knowledge, making the development process quicker compared to traditional protein-targeted drugs, which enhances their potential for therapeutic applications.
Casimersen (AMONDYS 45™): An Antisense Oligonucleotide for Duchenne Muscular Dystrophy.
Biomedicines
April 2024
Division of Clinical Sciences, College of Osteopathic Medicine, Marian University, Indianapolis, IN 46222, USA.
Casimersen (AMONDYS 45) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose gene mutation is amenable to exon 45 skipping. Administered intravenously, casimersen binds to the pre-mRNA of the gene to skip a mutated region of an exon, thereby producing an internally truncated yet functional dystrophin protein in DMD patients.
View Article and Find Full Text PDFInhibition of survivin by 2'--methyl phosphorothioate-modified steric-blocking antisense oligonucleotides.
RSC Adv
April 2024
Centre for Molecular Medicine and Innovative Therapeutics, Health Futures Institute, Murdoch University Murdoch 6150 Australia
Article Synopsis
- Chemically modified antisense oligonucleotides (ASOs) have been proven effective in treating various diseases, with ten approved ASO drugs currently on the market, mainly for conditions other than cancer.
- Researchers have developed steric-blocking ASOs that specifically target a known oncogene, initially screening seven candidates in HepG2 cells and identifying ASO-2 and ASO-7 as the most effective in reducing mRNA levels.
- Further testing demonstrated that ASO-7 not only inhibited mRNA expression in a dose-dependent manner but also significantly reduced survivin protein levels, suggesting its potential as a therapeutic BIRC5 inhibitor.
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