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Wang M, Birnkrant DJ, Super DM, Jacobs IB, Bahler RC. Progressive left ventricular dysfunction and long-term outcomes in patients with…

Pandya S, James KA, Westfield C, et al. Health profile of a cohort of adults with Duchenne muscular dystrophy. Muscle…

Siemionow M, Cwykiel J, Heydemann A, Garcia-Martinez J, Siemionow K, Szilagyi E. Creation of Dystrophin Expressing Chimeric Cells of Myoblast…

Ricotti V, Muntoni F, Voit T. Challenges of clinical trial design for DMD. Neuromuscular disorders : NMD 2015;25:932-5 Including a…

Min YL, Bassel-Duby R, Olson EN. CRISPR Correction of Duchenne Muscular Dystrophy. Annual review of medicine 2019;70:239-255   Abstract The…

Majowicz A, Salas D, Zabaleta N, et al. Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by…

Lim KRQ, Yoon C, Yokota T. Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy. Journal of personalized medicine…

Hakim CH, Wasala NB, Nelson CE, et al. AAV CRISPR editing rescues cardiac and muscle function for 18 months in…

Barthelemy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscular disorders : NMD 2018;28:803-824   Abstract…

Ebrahimi-Fakhari D, Dillmann U, Flotats-Bastardas M, et al. Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne…