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 WittPanayiotis Yiallouros, Zorica Zivkovic, Claudia E Kuehni, Jane S Lucas

Research output: Contribution to journalArticleAcademicpeer-review


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.

Original languageEnglish
JournalBMJ Open
Issue number3
Publication statusPublished - Jul 2020

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