Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial].

BORIS DOI
Publisher DOI
PubMed ID
32964005
Description
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.
Date of Publication
2020-07
Publication Type
Article
Subject(s)
600 - Technology::610 - Medicine & health
300 - Social sciences, sociology & anthropology::360 - Social problems & social services
Language(s)
en
Contributor(s)
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
Lucas, Jane S
Additional Credits
Institut für Sozial- und Präventivmedizin (ISPM)
Series
ERJ Open Research
Publisher
European Respiratory Society
ISSN
2312-0541
Access(Rights)
open.access
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