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

Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: European Respiratory Society 2020
Materias:
Editorials
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7487359/
https://www.ncbi.nlm.nih.gov/pubmed/32964005
http://dx.doi.org/10.1183/23120541.00377-2020

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