Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study

Erik Landfeldta IQVIA, Stockholm, SwedenCorrespondence[email protected]

https://orcid.org/0000-0002-5758-7151

Rongrong Zhangb PTC Therapeutics Sweden AB, Askim, Sweden

Anne-Marie Childsc Leeds Teaching Hospitals, Leeds, UK

Jessika Johannsend Department of Pediatrics, Neuropediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

Declan O'Rourkee Department of Neurology, Children’s Health Ireland at Temple Street, Dublin, Ireland

Thomas Sejersenf Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden;g Department of Neuropediatrics, Astrid Lindgren Children´s Hospital, Karolinska University Hospital, Stockholm, Sweden

Jurgis Strautmanish Department of Neurology and Neurosurgery, Children's Clinical University Hospital, Riga, Latvia

Ulrike Schara-Schmidti Department of Neuropediatrics, Developmental Neurology and Social Pediatrics, Centre for Neuromuscular Disorders in Children and Adolescents, Children´s University Clinic Essen, University of Duisburg-Essen, Essen, Germany

Mar Tuliniusj Department of Pediatrics, University of Gothenburg, Gothenburg, Sweden

Maggie C. Walterk Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany

Tracey Willisl Robert Jones and Agnes Hunt Hospital, Oswestry, UK

Katharina Bueschm PTC Therapeutics Switzerland, Zug, Switzerland

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Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study

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Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study

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