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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. Among authors: miller nf. 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.
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK. Mendell JR, et al. Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6. Mol Ther. 2017. PMID: 28279643 Free PMC article.
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Mendell JR, et al. Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19. Hum Gene Ther. 2019. PMID: 30838895 Free PMC article.
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
Waldrop MA, Karingada C, Storey MA, Powers B, Iammarino MA, Miller NF, Alfano LN, Noritz G, Rossman I, Ginsberg M, Mosher KA, Broomall E, Goldstein J, Bass N, Lowes LP, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Among authors: miller nf. Pediatrics. 2020 Sep;146(3):e20200729. doi: 10.1542/peds.2020-0729. Pediatrics. 2020. PMID: 32843442
Conference report on contractures in musculoskeletal and neurological conditions.
Nuckolls GH, Kinnett K, Dayanidhi S, Domenighetti AA, Duong T, Hathout Y, Lawlor MW, Lee SSM, Magnusson SP, McDonald CM, McNally EM, Miller NF, Olwin BB, Raghavan P, Roberts TJ, Rutkove SB, Sarwark JF, Senesac CR, Vogel LF, Walter GA, Willcocks RJ, Rymer WZ, Lieber RL. Nuckolls GH, et al. Among authors: miller nf. Muscle Nerve. 2020 Jun;61(6):740-744. doi: 10.1002/mus.26845. Epub 2020 Mar 7. Muscle Nerve. 2020. PMID: 32108365 Free PMC article. Review.
73 results