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Page 1
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC. Goemans NM, et al. Among authors: campion gv. N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. N Engl J Med. 2011. PMID: 21428760 Free 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.
Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne.
Lourbakos A, Yau N, de Bruijn P, Hiller M, Kozaczynska K, Jean-Baptiste R, Reza M, Wolterbeek R, Koeks Z, Ayoglu B, de Klerk D, Campion G, Zaharieva I, Nadarajah VD, Nilsson P, Al-Khalili Szigyarto C, Muntoni F, Lochmüller H, Verschuuren JJ, Goemans N, Tulinius M, Niks EH, de Kimpe S, Aartsma-Rus A, 't Hoen PAC, Spitali P. Lourbakos A, et al. Sci Rep. 2017 Dec 20;7(1):17888. doi: 10.1038/s41598-017-17982-y. Sci Rep. 2017. PMID: 29263366 Free PMC article.
A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy.
Beekman C, Sipkens JA, Testerink J, Giannakopoulos S, Kreuger D, van Deutekom JC, Campion GV, de Kimpe SJ, Lourbakos A. Beekman C, et al. Among authors: campion gv. PLoS One. 2014 Sep 22;9(9):e107494. doi: 10.1371/journal.pone.0107494. eCollection 2014. PLoS One. 2014. PMID: 25244123 Free PMC 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.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
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.
34 results