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Duchenne muscular dystrophy.
Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duan D, et al. Nat Rev Dis Primers. 2021 Feb 18;7(1):13. doi: 10.1038/s41572-021-00248-3. Nat Rev Dis Primers. 2021. PMID: 33602943 Free PMC article. Review.
Duplications in the DMD gene.
White SJ, Aartsma-Rus A, Flanigan KM, Weiss RB, Kneppers AL, Lalic T, Janson AA, Ginjaar HB, Breuning MH, den Dunnen JT. White SJ, et al. Hum Mutat. 2006 Sep;27(9):938-45. doi: 10.1002/humu.20367. Hum Mutat. 2006. PMID: 16917894
Local dystrophin restoration with antisense oligonucleotide PRO051.
van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, de Kimpe SJ, Ekhart PF, Venneker EH, Platenburg GJ, Verschuuren JJ, van Ommen GJ. van Deutekom JC, et al. N Engl J Med. 2007 Dec 27;357(26):2677-86. doi: 10.1056/NEJMoa073108. N Engl J Med. 2007. PMID: 18160687 Free article. Clinical Trial.
253 results