Art

J-GLOBAL ID：201702261209987457   Reference number：17A1545442

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

ナンセンス変異Duchenne型筋ジストロフィー(ACT DMD)患者のアタルレン(Ataluren):多施設,無作為化,二重盲検,プラセボ対照,第3相試験【Powered by NICT】

Author (32)： McDonald Craig M (University of California Davis School of Medicine, Davis, Sacramento, CA, USA) ,  Campbell Craig (Schulich School of Medicine and Dentistry, Western University, London, ON, Canada) ,  Torricelli Ricardo Erazo (Hospital Luis Calvo Mackenna, Santiago de Chile, Chile) ,  Finkel Richard S (Children’s Hospital of Philadelphia, Philadelphia, PA, USA) ,  Finkel Richard S (Nemours Children’s Hospital, Orlando, FL, USA) ,  Flanigan Kevin M (Nationwide Children’s Hospital, Columbus, OH, USA) ,  Goemans Nathalie (University Hospitals Leuven, KU Leuven, Belgium) ,  Heydemann Peter (Rush University Medical Center, Chicago, IL, USA) ,  Kaminska Anna (Medical University of Warsaw, Warsaw, Poland) ,  Kirschner Janbernd (Medical Center-University of Freiburg, University of Freiberg, Freiberg, Germany) ,  Muntoni Francesco (University College London Great Ormond Street Institute of Child Health, London, UK) ,  Osorio Andres Nascimento (Hospital Sant Joan de Deu, Universidad de Barcelona, CIBER, ISCIII, Barcelona, Spain) ,  Schara Ulrike (University Hospital Essen, University of Duisburg-Essen, Essen, Germany) ,  Sejersen Thomas (Karolinska University Hospital, Karolinska Institutet, Stockholm, Sweden) ,  Shieh Perry B (University of California, Los Angeles, CA, USA) ,  Sweeney H Lee (University of Florida, Gainesville, FL, USA) ,  Topaloglu Haluk (Hacettepe Children’s Hospital, Ankara, Turkey) ,  Tulinius Mar (Gothenburg University, Queen Silvia Children’s Hospital, Gothenburg, Sweden) ,  Vilchez Juan J (Hospital Universitario y Politecnico La Fe, CIBERER, Valencia, Spain) ,  Voit Thomas (University College London Great Ormond Street Institute of Child Health, London, UK) ,  Voit Thomas (National Institute for Health Research Great Ormond Street Hospital University College London Biomedical Research Centre, London, UK) ,  Wong Brenda (Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA) ,  Elfring Gary (PTC Therapeutics, South Plainfield, NJ, USA) ,  Kroger Hans (PTC Therapeutics, South Plainfield, NJ, USA) ,  Luo Xiaohui (PTC Therapeutics, South Plainfield, NJ, USA) ,  McIntosh Joseph (PTC Therapeutics, South Plainfield, NJ, USA) ,  Ong Tuyen (PTC Therapeutics, South Plainfield, NJ, USA) ,  Riebling Peter (PTC Therapeutics, South Plainfield, NJ, USA) ,  Souza Marcio (PTC Therapeutics, South Plainfield, NJ, USA) ,  Spiegel Robert J (PTC Therapeutics, South Plainfield, NJ, USA) ,  Peltz Stuart W (PTC Therapeutics, South Plainfield, NJ, USA) ,  Mercuri Eugenio (Department of Pediatric Neurology, Catholic University, Rome, Italy)
Material： Lancet
Volume： 390  Issue： 10101  Page： 1489-1498  Publication year： 2017 
JST Material Number： B0673A  ISSN： 0140-6736  CODEN： LANCA  Document type： Article
Article type： 原著論文  Country of issue： Netherlands (NLD)  Language： ENGLISH (EN)

Abstract/Point： Duchenne muscular dystrophy (D...

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Thesaurus term： forecast ,  base line ,  therapy ,  *double blind test ,  least squares method ,  hierarchical structure ,  Europe ,  oral administration ,  *placebo ,  *patient ,  evaluation criterion ,  adrenocortical hormone ,  Safety ,  *phase 3 study ,  Web (information system)

JST classification (2)： Clinical neurology in general  (GN01030H) ,  Drug therapy(=pharmacotherapy)for nervous system diseases  (GN05020Y)

Terms in the title (9)： ナンセンス変異 ,  Duchenne型筋ジストロフィー ,  ACT ,  患者 ,  施設 ,  二重盲検 ,  プラセボ ,  対照 ,  第3相試験