Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial].

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; ... (2020). Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial]. ERJ Open Research, 6(3), 00377-2020. European Respiratory Society 10.1183/23120541.00377-2020

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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.

Item Type:

Journal Article (Further Contribution)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine
04 Faculty of Medicine > Pre-clinic Human Medicine > Institute of Social and Preventive Medicine (ISPM)
04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine > Paediatric Pneumology

UniBE Contributor:

Kühni, Claudia

Subjects:

600 Technology > 610 Medicine & health
300 Social sciences, sociology & anthropology > 360 Social problems & social services

ISSN:

2312-0541

Publisher:

European Respiratory Society

Funders:

[4] Swiss National Science Foundation

Language:

English

Submitter:

Andrea Flükiger-Flückiger

Date Deposited:

15 Oct 2020 20:01

Last Modified:

11 Mar 2021 21:00

Publisher DOI:

10.1183/23120541.00377-2020

PubMed ID:

32964005

BORIS DOI:

10.7892/boris.146860

URI:

https://boris.unibe.ch/id/eprint/146860

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