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Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study.
Longo, Nicola; Zori, Roberto; Wasserstein, Melissa P; Vockley, Jerry; Burton, Barbara K; Decker, Celeste; Li, Mingjin; Lau, Kelly; Jiang, Joy; Larimore, Kevin; Thomas, Janet A.
Afiliación
  • Longo N; Department of Pediatrics, Division of Medical Genetics, University of Utah, 295 Chipeta Way, Salt Lake City, UT, 84108, USA. nicola.longo@hsc.utah.edu.
  • Zori R; Division of Genetics and Metabolism, University of Florida, PO Box 100296 UFHSC, Gainesville, FL, 32610, USA.
  • Wasserstein MP; Department of Pediatrics, The Children's Hospital at Montefiore, 3415 Bainbridge Ave, Bronx, NY, 10467, USA.
  • Vockley J; Department of Pediatrics, Division of Medical Genetics, University of Pittsburgh and Children's Hospital of Pittsburgh, 4401 Penn Avenue, Pittsburgh, PA, 15224, USA.
  • Burton BK; Department of Pediatrics, Division of Genetics, Birth Defects & Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, 225 E. Chicago Ave, Chicago, IL, 60611, USA.
  • Decker C; BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA.
  • Li M; BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA.
  • Lau K; BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA.
  • Jiang J; BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA.
  • Larimore K; BioMarin Pharmaceutical Inc, 105 Digital Drive, Novato, CA, 94949, USA.
  • Thomas JA; Department of Pediatrics, Section of Clinical Genetics and Metabolism, University of Colorado Hospital, 12605 E. 16th St, Aurora, CO, 80045, USA.
Orphanet J Rare Dis ; 13(1): 108, 2018 07 04.
Article en En | MEDLINE | ID: mdl-29973227
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenilanina Amoníaco-Liasa / Fenilcetonurias / Proteínas Recombinantes Límite: Humans Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenilanina Amoníaco-Liasa / Fenilcetonurias / Proteínas Recombinantes Límite: Humans Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido