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5 p, 611.5 KB Access to medicines for rare diseases : beating the drum for primary ciliary dyskinesia / Crowley, Suzanne (Oslo University Hospital (Oslo, Noruega)) ; Azevedo, Ines (Universidade do Porto) ; Boon, Mieke (University Hospitals Gasthuisberg (Leuven, Bélgica)) ; Bush, Andrew (Royal Brompton Hospital (Londres)) ; Eber, Ernst (Medical University of Graz) ; Haarman, Eric (Amsterdam UMC. University Medical Center) ; Karadag, Bulent (Marmara University) ; Kötz, Karsten (Sahlgrenska University Hospital (Suècia)) ; Leigh, Margaret (University of North Carolina at Chapel Hill) ; Moreno Galdó, Antonio (Universitat Autònoma de Barcelona. Departament de Pediatria, Obstetrícia i Ginecologia i de Medicina Preventiva i Salut Pública) ; Mussaffi, Huda (Tel Aviv University) ; Nielsen, Kim G. ; Omran, Heymut (University Hospital Muenster (Alemanya)) ; Papon, Jean-François (Hôpital Kremlin-Bicetre) ; Pohunek, Petr (Charles University. Faculty of Medicine in Hradec Králové) ; Priftis, Kostas (University General Hospital Attikon (Haidari, Grècia)) ; Rindlisbacher, Bernhard (Patient Association Kartagener Syndrom und Primäre Ciliäre Dyskinesie e.V., Steffisburg, Switzerland) ; Santamaria, Francesca (Federico II University) ; Valiulis, Arunas (European Academy of Paediatrics (Bélgica)) ; Witt, Michal (Institute of Human Genetics Polish Academy of Sciences(Polònia)) ; Yiallouros, Panayiotis (University of Cyprus) ; Zivkovic, Zorica (Novi Sad (Sèrbia)) ; Kuehni, Claudia E. (University Children's Hospital Bern (Suïssa)) ; Lucas, Jane S. (University Hospital Southampton NHS Foundation Trust (Regne Unit))
Rare diseases are collectively common, affecting an estimated 6. 2% of the world's population [1], but each rare disease affects fewer than 4 to 5 in 10 000 individuals in Europe or less than 200 000 individuals in the USA [2]. [...]
2020 - 10.1183/23120541.00377-2020
ERJ Open Research, Vol. 6 (september 2020)  

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