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An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.
Kazi, Zoheb B; Desai, Ankit K; Troxler, R Bradley; Kronn, David; Packman, Seymour; Sabbadini, Marta; Rizzo, William B; Scherer, Katalin; Abdul-Rahman, Omar; Tanpaiboon, Pranoot; Nampoothiri, Sheela; Gupta, Neerja; Feigenbaum, Annette; Niyazov, Dmitriy M; Sherry, Langston; Segel, Reeval; McVie-Wylie, Alison; Sung, Crystal; Joseph, Alexandra M; Richards, Susan; Kishnani, Priya S.
Afiliación
  • Kazi ZB; Division of Medical Genetics, Department of Pediatrics, Duke University Health System, Durham, NC, USA.
  • Desai AK; Division of Medical Genetics, Department of Pediatrics, Duke University Health System, Durham, NC, USA.
  • Troxler RB; Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, USA.
  • Kronn D; Department of Pediatrics, New York Medical College, Valhalla, NY, USA.
  • Packman S; Division of Medical Genetics, Department of Pediatrics, University of California, San Francisco, CA, USA.
  • Sabbadini M; Division of Medical Genetics, Department of Pediatrics, University of California, San Francisco, CA, USA.
  • Rizzo WB; Department of Pediatrics, University of Nebraska Medical Center, Nebraska Medical Center Omaha, Omaha, NE, USA.
  • Scherer K; Department of Neurology, University of Arizona, Tucson, AZ, USA.
  • Abdul-Rahman O; Munroe-Meyer Institute for Genetics and Rehabilitation, University of Nebraska Medical Center, Omaha, NE, USA.
  • Tanpaiboon P; Quest Diagnostics and Children's National Health System, Washington, DC, USA.
  • Nampoothiri S; Amrita Institute of Medical Sciences & Research Centre, Kochi, Kerala, India.
  • Gupta N; All India Institute of Medical Sciences, New Delhi, India.
  • Feigenbaum A; University of California, San Diego, CA, USA.
  • Niyazov DM; Department of Pediatrics, Ochsner Health System, New Orleans, LA, USA.
  • Sherry L; Department of Pediatrics, Ochsner Health System, New Orleans, LA, USA.
  • Segel R; Medical Genetics Institute, Shaare Zedek Medical Center and the Hebrew University School of Medicine, Jerusalem, Israel.
  • McVie-Wylie A; Sanofi Genzyme, Framingham, MA, USA.
  • Sung C; Sanofi Genzyme, Framingham, MA, USA.
  • Joseph AM; Sanofi Genzyme, Framingham, MA, USA.
  • Richards S; Sanofi Genzyme, Framingham, MA, USA.
  • Kishnani PS; Division of Medical Genetics, Department of Pediatrics, Duke University Health System, Durham, NC, USA. priya.kishnani@duke.edu.
Genet Med ; 21(4): 887-895, 2019 04.
Article en En | MEDLINE | ID: mdl-30214072
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Metotrexato / Tolerancia Inmunológica Tipo de estudio: Observational_studies Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2019 Tipo del documento: Article
Texto completo
Imprimir
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Metotrexato / Tolerancia Inmunológica Tipo de estudio: Observational_studies Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2019 Tipo del documento: Article