Medizinische Universität Graz - Research portal
Selected Publication:
SHR Neuro Cancer Cardio Lipid Metab Microb
Crowley, S; Azevedo, I; Boon, M; Bush, A; Eber, E; Haarman, E; Karadag, B; Kötz, K; Leigh, M; Moreno-Galdó, A; Mussaffi, H; Nielsen, KG; Omran, H; Papon, JF; Pohunek, P; Priftis, K; Rindlisbacher, B; Santamaria, F; Valiulis, A; Witt, M; Yiallouros, P; Zivkovic, Z; Kuehni, CE; Lucas, JS.
Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia.
ERJ Open Res. 2020; 6(3):
Doi: 10.1183/23120541.00377-2020
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- Co-authors Med Uni Graz
- Eber Ernst
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- 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. Copyright ©ERS 2020.