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J-GLOBAL ID：201702261209987457 整理番号：17A1545442

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】

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

著者 (32件)：

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