Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia
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.
Crowley, Suzanne
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Ines, Azevedo
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Boon, Mieke
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Bush, Andrew
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Eber, Ernst
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Lucas, Jane
5cb3546c-87b2-4e59-af48-402076e25313
14 September 2020
Crowley, Suzanne
41080327-ab5c-4a5d-8770-37d55278c130
Ines, Azevedo
f3eed006-f9de-4a34-b88a-580946d45ae6
Boon, Mieke
31b7dcb0-a53f-4e56-81d8-df0883f97370
Bush, Andrew
ea288e1f-bd13-4422-be1c-cac640edb239
Eber, Ernst
9de771c4-6a52-4751-8219-b20cd9ee83fd
Lucas, Jane
5cb3546c-87b2-4e59-af48-402076e25313
Crowley, Suzanne, Ines, Azevedo, Boon, Mieke, Bush, Andrew, Eber, Ernst and Lucas, Jane
(2020)
Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia.
ERJ Open Research, 6 (3), [00377-2020].
(doi:10.1183/23120541.00377-2020).
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.
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Accepted/In Press date: 8 July 2020
Published date: 14 September 2020
Additional Information:
Copyright ©ERS 2020.
Identifiers
Local EPrints ID: 442793
URI: http://eprints.soton.ac.uk/id/eprint/442793
ISSN: 2312-0541
PURE UUID: 9ac07d40-ca0c-43ee-a0db-b4e19a062480
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Date deposited: 27 Jul 2020 16:31
Last modified: 17 Mar 2024 02:55
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Contributors
Author:
Suzanne Crowley
Author:
Azevedo Ines
Author:
Mieke Boon
Author:
Andrew Bush
Author:
Ernst Eber
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