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

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

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.

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Veröffentlicht in:ERJ OPEN RESEARCH 2020-07, Vol.6 (3)
Hauptverfasser: Crowley, Suzanne, Azevedo, Ines, Boon, Mieke, Bush, Andrew, Eber, Ernst, Haarman, Eric, Karadag, Bulent, Kotz, Karsten, Leigh, Margaret, Moreno-Galdo, Antonio, Mussaffi, Huda, Nielsen, Kim G, Omran, Heymut, Papon, Jean-Francois, Pohunek, Petr, Priftis, Kostas, Rindlisbacher, Bernhard, Santamaria, Francesca, Valiulis, Arunas, Witt, Michal, Yiallouros, Panayiotis, Zivkovic, Zorica, Kuehni, Claudia E, Lucas, Jane S
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Sprache:eng
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Zusammenfassung: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.
ISSN:2312-0541
2312-0541