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34 results

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Single Ascending Dose Study of a Short Interfering RNA Targeting Lipoprotein(a) Production in Individuals With Elevated Plasma Lipoprotein(a) Levels.
Nissen SE, Wolski K, Balog C, Swerdlow DI, Scrimgeour AC, Rambaran C, Wilson RJ, Boyce M, Ray KK, Cho L, Watts GF, Koren M, Turner T, Stroes ES, Melgaard C, Campion GV. Nissen SE, et al. Among authors: campion gv. JAMA. 2022 May 3;327(17):1679-1687. doi: 10.1001/jama.2022.5050. JAMA. 2022. PMID: 35368052 Free PMC article. Clinical Trial.
Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids.
Goemans N, Tulinius M, Kroksmark AK, Wilson R, van den Hauwe M, Campion G. Goemans N, et al. Neuromuscul Disord. 2017 Mar;27(3):203-213. doi: 10.1016/j.nmd.2016.11.013. Epub 2016 Nov 25. Neuromuscul Disord. 2017. PMID: 28169120 Free article.
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, Liefaard L, Lourbakos A, Morgan A, Nakielny J, Quarcoo N, Ricotti V, Rolfe K, Servais L, Wardell C, Wilson R, Wright P, Kraus JE. Voit T, et al. Lancet Neurol. 2014 Oct;13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4. Epub 2014 Sep 7. Lancet Neurol. 2014. PMID: 25209738 Clinical Trial.
Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.
McDonald CM, Wong B, Flanigan KM, Wilson R, de Kimpe S, Lourbakos A, Lin Z, Campion G; DEMAND V study group. McDonald CM, et al. Ann Clin Transl Neurol. 2018 Jul 11;5(8):913-926. doi: 10.1002/acn3.579. eCollection 2018 Aug. Ann Clin Transl Neurol. 2018. PMID: 30128316 Free PMC article.
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.
Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, Kraus JE, Lourbakos A, Lin Z, Campion G, Wang SX, Campbell C; DEMAND III study group. Goemans N, et al. Neuromuscul Disord. 2018 Jan;28(1):4-15. doi: 10.1016/j.nmd.2017.10.004. Epub 2017 Dec 6. Neuromuscul Disord. 2018. PMID: 29203355 Free article. Clinical Trial.
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial.
Flanigan KM, Voit T, Rosales XQ, Servais L, Kraus JE, Wardell C, Morgan A, Dorricott S, Nakielny J, Quarcoo N, Liefaard L, Drury T, Campion G, Wright P. Flanigan KM, et al. Neuromuscul Disord. 2014 Jan;24(1):16-24. doi: 10.1016/j.nmd.2013.09.004. Epub 2013 Sep 11. Neuromuscul Disord. 2014. PMID: 24321374 Free PMC article. Clinical Trial.
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