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Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy.
Willcocks, Rebecca J; Forbes, Sean C; Walter, Glenn A; Sweeney, Lee; Rodino-Klapac, Louise R; Mendell, Jerry R; Vandenborne, Krista.
Afiliação
  • Willcocks RJ; Department of Physical Therapy, University of Florida, Gainesville.
  • Forbes SC; Department of Physical Therapy, University of Florida, Gainesville.
  • Walter GA; Department of Physical Therapy, University of Florida, Gainesville.
  • Sweeney L; Department of Physical Therapy, University of Florida, Gainesville.
  • Rodino-Klapac LR; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
  • Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
  • Vandenborne K; Department of Physical Therapy, University of Florida, Gainesville.
JAMA Netw Open ; 4(1): e2031851, 2021 01 04.
Article em En | MEDLINE | ID: mdl-33394000

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Imageamento por Ressonância Magnética / Terapia Genética / Distrofina / Distrofia Muscular de Duchenne Tipo de estudo: Observational_studies Limite: Child / Child, preschool / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Imageamento por Ressonância Magnética / Terapia Genética / Distrofina / Distrofia Muscular de Duchenne Tipo de estudo: Observational_studies Limite: Child / Child, preschool / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article