Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial].
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BORIS DOI
Date of Publication
July 2020
Publication Type
Article
Division/Institute
Author
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 |
Series
ERJ Open Research
ISSN or ISBN (if monograph)
2312-0541
Publisher
European Respiratory Society
Language
English
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.
File(s)
| File | File Type | Format | Size | License | Publisher/Copright statement | Content | |
|---|---|---|---|---|---|---|---|
| Crowley ERJOpenRes 2020.pdf | text | Adobe PDF | 611.55 KB | published |