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Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.
Deng, Callie; Zhao, Peter Y; Branham, Kari; Schlegel, Dana; Fahim, Abigail T; Jayasundera, Thiran K; Khan, Naheed; Besirli, Cagri G.
Afiliação
  • Deng C; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Zhao PY; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Branham K; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Schlegel D; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Fahim AT; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Jayasundera TK; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Khan N; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA.
  • Besirli CG; Department of Ophthalmology and Visual Sciences, University of Michigan W.K. Kellogg Eye Center, Ann Arbor, MI, USA. cbesirli@umich.edu.
Graefes Arch Clin Exp Ophthalmol ; 260(5): 1543-1550, 2022 May.
Article em En | MEDLINE | ID: mdl-35001204

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Amaurose Congênita de Leber Tipo de estudo: Diagnostic_studies / Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: Graefes Arch Clin Exp Ophthalmol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Amaurose Congênita de Leber Tipo de estudo: Diagnostic_studies / Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: Graefes Arch Clin Exp Ophthalmol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos