Your browser doesn't support javascript.
loading
Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia.
Crowley, Suzanne; Azevedo, Inês; Boon, Mieke; Bush, Andrew; Eber, Ernst; Haarman, Eric; Karadag, Bulent; Kötz, Karsten; Leigh, Margaret; Moreno-Galdó, Antonio; Mussaffi, Huda; Nielsen, Kim G; Omran, Heymut; Papon, Jean-François; Pohunek, Petr; Priftis, Kostas; Rindlisbacher, Bernhard; Santamaria, Francesca; Valiulis, Arunas; Witt, Michal; Yiallouros, Panayiotis; Zivkovic, Zorica; Kuehni, Claudia E; Lucas, Jane S.
Afiliación
  • Crowley S; Paediatric Dept for Lung and Allergic diseases, Oslo University Hospital, Oslo, Norway.
  • Azevedo I; Centro Materno-Pediátrico, Centro Hospitalar Universitário de S. João, Porto, Portugal.
  • Boon M; Departamento de Ginecologia-Obstetrícia e Pediatria, Faculdade de Medicina, Universidade do Porto, Porto, Portugal.
  • Bush A; Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium.
  • Eber E; Depts of Paediatrics and Paediatric Respiratory Medicine, Imperial College and Royal Brompton Hospital, London, UK.
  • Haarman E; Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
  • Karadag B; Dept of Pediatric Pulmonology, VU University Medical Center, Amsterdam, The Netherlands.
  • Kötz K; Dept of Pediatric Pulmonology, Marmara University, School of Medicine, Istanbul, Turkey.
  • Leigh M; Queen Silvias Children's Hospital, Sahlgrenska University Hospital, Gothenburg, Sweden.
  • Moreno-Galdó A; Dept of Pediatrics and Marsico Lung Institute, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Mussaffi H; Pediatric Pulmonology Section, Hospital Universitari Vall d'Hebron, Barcelona, Spain.
  • Nielsen KG; Universitat Autònoma de Barcelona, CIBERER, Barcelona, Spain.
  • Omran H; Schneider Children's Medical Center of Israel, Petach-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
  • Papon JF; Danish PCD Centre, Pediatric Pulmonary Service, Dept of Pediatrics and Adolescent Medicine, Rigshospitalet (Copenhagen University Hospital), Copenhagen, Denmark.
  • Pohunek P; Dept of General Pediatrics, University Hospital, Westfalian Wilhelms-University, Muenster, Germany.
  • Priftis K; 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.
  • Rindlisbacher B; Paediatric Dept, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic.
  • Santamaria F; 3rd Dept of Paediatrics, University General Hospital Attikon, National and Kapodistrian University of Athens, Athens, Greece.
  • Valiulis A; Patient Association Kartagener Syndrom und Primäre Ciliäre Dyskinesie e.V., Steffisburg, Switzerland.
  • Witt M; Pediatric Pulmonology, Dept of Translational Medical Sciences, Federico II University, Azienda Ospedaliera Universitaria Federico II, Naples, Italy.
  • Yiallouros P; Vilnius University Medical Faculty, Institute of Clinical Medicine, Clinic of Children's Diseases, Vilnius, Lithuania.
  • Zivkovic Z; European Academy of Paediatrics (EAP/UEMS-SP), Brussels, Belgium.
  • Kuehni CE; Dept of Molecular and Clinical Genetics, Institute of Human Genetics Polish Academy of Sciences, Poznan, Poland.
  • Lucas JS; Medical School, University of Cyprus, Nicosia, Cyprus.
ERJ Open Res ; 6(3)2020 Jul.
Article en En | MEDLINE | ID: mdl-32964005
ABSTRACT
Primary ciliary dyskinesia, a rare disease causing bronchiectasis, lacks a sound evidence base for treatment. @beatpcd proposes 1) forming a PCD European clinical trial network to address this situation and 2) conducting n-of-1 trials to access medication. https//bit.ly/3j5blfM.
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: ERJ Open Res Año: 2020 Tipo del documento: Article País de afiliación: Noruega
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: ERJ Open Res Año: 2020 Tipo del documento: Article País de afiliación: Noruega