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Journal of Medical Economics Volume 25, 2022 - Issue 1
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Genetic Disease

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]
ORCID Iconhttps://orcid.org/0000-0002-5758-7151
ORCID Icon,
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
 show all
Pages 808-816 | Received 15 Apr 2022, Accepted 31 May 2022, Published online: 14 Jun 2022

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