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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Mendell, Jerry R; Sahenk, Zarife; Lehman, Kelly; Nease, Carrie; Lowes, Linda P; Miller, Natalie F; Iammarino, Megan A; Alfano, Lindsay N; Nicholl, Amanda; Al-Zaidy, Samiah; Lewis, Sarah; Church, Kathleen; Shell, Richard; Cripe, Linda H; Potter, Rachael A; Griffin, Danielle A; Pozsgai, Eric; Dugar, Ashish; Hogan, Mark; Rodino-Klapac, Louise R.
Affiliation
  • Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Sahenk Z; Department of Pediatrics, The Ohio State University, Columbus.
  • Lehman K; Department of Neurology, The Ohio State University, Columbus.
  • Nease C; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Lowes LP; Department of Pediatrics, The Ohio State University, Columbus.
  • Miller NF; Department of Neurology, The Ohio State University, Columbus.
  • Iammarino MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Alfano LN; Department of Pediatrics, The Ohio State University, Columbus.
  • Nicholl A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Al-Zaidy S; Department of Pediatrics, The Ohio State University, Columbus.
  • Lewis S; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Church K; Department of Pediatrics, The Ohio State University, Columbus.
  • Shell R; Department of Neurology, The Ohio State University, Columbus.
  • Cripe LH; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Potter RA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Griffin DA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Pozsgai E; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Dugar A; Department of Pediatrics, The Ohio State University, Columbus.
  • Hogan M; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Rodino-Klapac LR; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
JAMA Neurol ; 77(9): 1122-1131, 2020 09 01.
Article in En | MEDLINE | ID: mdl-32539076
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Dystrophin / Outcome Assessment, Health Care / Muscular Dystrophy, Duchenne Type of study: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Humans / Male Language: En Year: 2020 Type: Article
Fulltext
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PubMed Links
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Full text: 1 Database: MEDLINE Main subject: Genetic Therapy / Dystrophin / Outcome Assessment, Health Care / Muscular Dystrophy, Duchenne Type of study: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Child / Child, preschool / Humans / Male Language: En Year: 2020 Type: Article