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, S., Azevedo, I., Boon, M., Bush, A., Eber, E., Haarman, E., Karadag, B., Kötz, K., Leigh, M., Moreno-Galdó, A., Mussaffi, H., Nielsen, K.G., Omran, H., Papon, J., Pohunek, P., Priftis, K., Rindlisbacher, B., Santamaria, F., Valiulis, A., Witt, M., et al.. - 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, FrancescaMembro 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
