Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia

Research output: Contribution to journalEditorialResearch

Documents

  • Suzanne Crowley
  • Inês Azevedo
  • Mieke Boon
  • Andrew Bush
  • Ernst Eber
  • Eric Haarman
  • Bulent Karadag
  • Karsten Kötz
  • Margaret Leigh
  • Antonio Moreno-Galdó
  • Huda Mussaffi
  • Heymut Omran
  • Jean-François Papon
  • Petr Pohunek
  • Kostas Priftis
  • Bernhard Rindlisbacher
  • Francesca Santamaria
  • Arunas Valiulis
  • Michal Witt
  • Panayiotis Yiallouros
  • Zorica Zivkovic
  • Claudia E Kuehni
  • Jane S Lucas

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.

Original languageEnglish
Article number00377-2020
Journal ERJ Open Research
Volume6
Issue number3
Number of pages5
ISSN2312-0541
DOIs
Publication statusPublished - 2020

ID: 261582395