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Alexa C. Klimchak, Lauren E. Sedita, Louise R. Rodino-Klapac, Jerry R. Mendell, Craig M. McDonald, Katherine L. Gooch & Daniel C. Malone. (2023) Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. Journal of Market Access & Health Policy 11:1.
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Arash Salmaninejad, Yousef Jafari Abarghan, Saeed Bozorg Qomi, Hadi Bayat, Meysam Yousefi, Sara Azhdari, Samaneh Talebi & Majid Mojarrad. (2021) Common therapeutic advances for Duchenne muscular dystrophy (DMD). International Journal of Neuroscience 131:4, pages 370-389.
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Tri Le Bao, Rakesh N. Veedu, Sue Fletcher & Steve D. Wilton. (2016) Antisense oligonucleotide development for the treatment of muscular dystrophies. Expert Opinion on Orphan Drugs 4:2, pages 139-152.
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Mohsan Iftikhar, Justin Frey, Md.Jasimuddin Shohan, Sohail Malek & Shaker A. Mousa. (2021) Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy. Pharmacology & Therapeutics 220, pages 107719.
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Antoine Boulanger Piette, Dounia Hamoudi, Laetitia Marcadet, Françoise Morin, Anteneh Argaw, Leanne Ward & Jérôme Frenette. (2018) Targeting the Muscle-Bone Unit: Filling Two Needs with One Deed in the Treatment of Duchenne Muscular Dystrophy. Current Osteoporosis Reports 16:5, pages 541-553.
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Mehrdad Dirin, Ernst Urban, Bodo Lachmann, Christian R Noe & Johannes Winkler. (2015) Concise postsynthetic preparation of oligonucleotide–oligopeptide conjugates through facile disulfide bond formation. Future Medicinal Chemistry 7:13, pages 1657-1673.
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Jon Tinsley, Neil Robinson & Kay E. Davies. (2015) Safety, tolerability, and pharmacokinetics of SMT C1100, a 2‐arylbenzoxazole utrophin modulator, following single‐ and multiple‐dose administration to healthy male adult volunteers. The Journal of Clinical Pharmacology 55:6, pages 698-707.
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Volker Baumann & Johannes Winkler. (2014) miRNA-based therapies: strategies and delivery platforms for oligonucleotide and non-oligonucleotide agents. Future Medicinal Chemistry 6:17, pages 1967-1984.
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