Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia
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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.
Original language | English |
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Article number | 00377-2020 |
Journal | ERJ Open Research |
Volume | 6 |
Issue number | 3 |
Number of pages | 5 |
ISSN | 2312-0541 |
DOIs | |
Publication status | Published - 2020 |
ID: 261582395