Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia

• Suzanne Crowley,
• Inês Azevedo,
• Mieke Boon,
• Andrew Bush,
• Ernst Eber,
• Eric Haarman,
• Bulent Karadag,
• Karsten Kötz,
• Margaret Leigh,
• Antonio Moreno-Galdó,
• Huda Mussaffi,
• Kim G. Nielsen,
• Heymut Omran,
• Jean-François Papon,
• Petr Pohunek,
• Kostas Priftis,
• Bernhard Rindlisbacher,
• Francesca Santamaria,
• Arunas Valiulis,
• Michal Witt,
• Panayiotis Yiallouros,
• Zorica Zivkovic,
• Claudia E. Kuehni,
• Jane S. Lucas

Affiliations

Suzanne Crowley

Paediatric Dept for Lung and Allergic diseases, Oslo University Hospital, Oslo, Norway

Inês Azevedo

Centro Materno-Pediátrico, Centro Hospitalar Universitário de S. João, Porto, Portugal

Mieke Boon

Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium

Andrew Bush

Depts of Paediatrics and Paediatric Respiratory Medicine, Imperial College and Royal Brompton Hospital, London, UK

Ernst Eber

Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria

Eric Haarman

Dept of Pediatric Pulmonology, VU University Medical Center, Amsterdam, The Netherlands

Bulent Karadag

Dept of Pediatric Pulmonology, Marmara University, School of Medicine, Istanbul, Turkey

Karsten Kötz

Queen Silvias Children's Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden

Margaret Leigh

Dept of Pediatrics and Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

Antonio Moreno-Galdó

Pediatric Pulmonology Section, Hospital Universitari Vall d'Hebron, Barcelona, Spain

Huda Mussaffi

Schneider Children's Medical Center of Israel, Petach-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel

Kim G. Nielsen

Danish PCD Centre, Pediatric Pulmonary Service, Dept of Pediatrics and Adolescent Medicine, Rigshospitalet (Copenhagen University Hospital), Copenhagen, Denmark

Heymut Omran

Dept of General Pediatrics, University Hospital, Westfalian Wilhelms-University, Muenster, Germany

Jean-François Papon

AP-HP, Hôpital Kremlin-Bicetre, Service d'ORL et de Chirurgie Cervico-Faciale and Faculté de Médecine, Université Paris-Saclay, 94070 Le Kremlin-Bicêtre, INSERM, U955 and CNRS, ERL 7240, Créteil, France

Petr Pohunek

Paediatric Dept, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic

Kostas Priftis

3rd Dept of Paediatrics, University General Hospital Attikon, National and Kapodistrian University of Athens, Athens, Greece

Bernhard Rindlisbacher

Patient Association Kartagener Syndrom und Primäre Ciliäre Dyskinesie e.V., Steffisburg, Switzerland

Francesca Santamaria

Pediatric Pulmonology, Dept of Translational Medical Sciences, Federico II University, Azienda Ospedaliera Universitaria Federico II, Naples, Italy

Arunas Valiulis

Vilnius University Medical Faculty, Institute of Clinical Medicine, Clinic of Children's Diseases, Vilnius, Lithuania

Michal Witt

Dept of Molecular and Clinical Genetics, Institute of Human Genetics Polish Academy of Sciences, Poznan, Poland

Panayiotis Yiallouros

Medical School, University of Cyprus, Nicosia, Cyprus

Zorica Zivkovic

Children's Hospital for Lung Diseases and TB, Medical Centre “Dr Dragisa Misovic”, Belgrade, Serbia

Claudia E. Kuehni

Institute of Social and Preventive Medicine and Paediatric Respiratory Medicine, Children's University Hospital of Bern, University of Bern, Bern, Switzerland

Jane S. Lucas

Primary Ciliary Dyskinesia Centre, University Hospital Southampton NHS Foundation Trust and Clinical and Experimental Medicine, University of Southampton, Southampton, UK