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.

Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia / 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; Kuehni, Claudia E; Lucas, Jane S. - In: ERJ OPEN RESEARCH. - ISSN 2312-0541. - 6:3(2020), pp. 00377-2020-00381-020. [10.1183/23120541.00377-2020]

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

Santamaria, Francesca
Membro del Collaboration Group
;
2020

Abstract

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.
2020
Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia / 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; Kuehni, Claudia E; Lucas, Jane S. - In: ERJ OPEN RESEARCH. - ISSN 2312-0541. - 6:3(2020), pp. 00377-2020-00381-020. [10.1183/23120541.00377-2020]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/825475
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