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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
McDonald, Craig M; Campbell, Craig; Torricelli, Ricardo Erazo; Finkel, Richard S; Flanigan, Kevin M; Goemans, Nathalie; Heydemann, Peter; Kaminska, Anna; Kirschner, Janbernd; Muntoni, Francesco; Osorio, Andrés Nascimento; Schara, Ulrike; Sejersen, Thomas; Shieh, Perry B; Sweeney, H Lee; Topaloglu, Haluk; Tulinius, Már; Vilchez, Juan J; Voit, Thomas; Wong, Brenda; Elfring, Gary; Kroger, Hans; Luo, Xiaohui; McIntosh, Joseph; Ong, Tuyen; Riebling, Peter; Souza, Marcio; Spiegel, Robert J; Peltz, Stuart W; Mercuri, Eugenio.
Afiliação
  • McDonald CM; University of California Davis School of Medicine, Davis, Sacramento, CA, USA. Electronic address: cmmcdonald@ucdavis.edu.
  • Campbell C; Schulich School of Medicine and Dentistry, Western University, London, ON, Canada.
  • Torricelli RE; Hospital Luis Calvo Mackenna, Santiago de Chile, Chile.
  • Finkel RS; Children's Hospital of Philadelphia, Philadelphia, PA, USA; Nemours Children's Hospital, Orlando, FL, USA.
  • Flanigan KM; Nationwide Children's Hospital, Columbus, OH, USA.
  • Goemans N; University Hospitals Leuven, KU Leuven, Belgium.
  • Heydemann P; Rush University Medical Center, Chicago, IL, USA.
  • Kaminska A; Medical University of Warsaw, Warsaw, Poland.
  • Kirschner J; Medical Center-University of Freiburg, University of Freiberg, Freiberg, Germany.
  • Muntoni F; University College London Great Ormond Street Institute of Child Health, London, UK.
  • Osorio AN; Hospital Sant Joan de Déu, Universidad de Barcelona, CIBER, ISCIII, Barcelona, Spain.
  • Schara U; University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
  • Sejersen T; Karolinska University Hospital, Karolinska Institutet, Stockholm, Sweden.
  • Shieh PB; University of California, Los Angeles, CA, USA.
  • Sweeney HL; University of Florida, Gainesville, FL, USA.
  • Topaloglu H; Hacettepe Children's Hospital, Ankara, Turkey.
  • Tulinius M; Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
  • Vilchez JJ; Hospital Universitario y Politécnico La Fe, CIBERER, Valencia, Spain.
  • Voit T; University College London Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research Great Ormond Street Hospital University College London Biomedical Research Centre, London, UK.
  • Wong B; Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Elfring G; PTC Therapeutics, South Plainfield, NJ, USA.
  • Kroger H; PTC Therapeutics, South Plainfield, NJ, USA.
  • Luo X; PTC Therapeutics, South Plainfield, NJ, USA.
  • McIntosh J; PTC Therapeutics, South Plainfield, NJ, USA.
  • Ong T; PTC Therapeutics, South Plainfield, NJ, USA.
  • Riebling P; PTC Therapeutics, South Plainfield, NJ, USA.
  • Souza M; PTC Therapeutics, South Plainfield, NJ, USA.
  • Spiegel RJ; PTC Therapeutics, South Plainfield, NJ, USA.
  • Peltz SW; PTC Therapeutics, South Plainfield, NJ, USA.
  • Mercuri E; Department of Pediatric Neurology, Catholic University, Rome, Italy.
Lancet ; 390(10101): 1489-1498, 2017 09 23.
Article em En | MEDLINE | ID: mdl-28728956
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Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oxidiazóis / Códon sem Sentido / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Child / Humans / Male Idioma: En Revista: Lancet Ano de publicação: 2017 Tipo de documento: Article
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oxidiazóis / Códon sem Sentido / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Child / Humans / Male Idioma: En Revista: Lancet Ano de publicação: 2017 Tipo de documento: Article