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Emerging drugs for Duchenne muscular dystrophy.
Malik V, Rodino-Klapac LR, Mendell JR. Malik V, et al. Among authors: mendell jr. Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965. Expert Opin Emerg Drugs. 2012. PMID: 22632414 Free PMC article. Review.
Duchenne muscular dystrophy: CRISPR/Cas9 treatment.
Mendell JR, Rodino-Klapac LR. Mendell JR, et al. Cell Res. 2016 May;26(5):513-4. doi: 10.1038/cr.2016.28. Epub 2016 Mar 1. Cell Res. 2016. PMID: 26926391 Free PMC article.
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Mendell JR, et al. JAMA Neurol. 2020 Sep 1;77(9):1122-1131. doi: 10.1001/jamaneurol.2020.1484. JAMA Neurol. 2020. PMID: 32539076 Free PMC article. Clinical Trial.
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.
Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR. Malik V, et al. Among authors: mendell jr. Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024. Ann Neurol. 2010. PMID: 20517938 Clinical Trial.
Dystrophin immunity in Duchenne's muscular dystrophy.
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Mendell JR, et al. N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228. N Engl J Med. 2010. PMID: 20925545 Free PMC article. Clinical Trial.
408 results