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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
| Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
European Respiratory Society
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7487359/ https://www.ncbi.nlm.nih.gov/pubmed/32964005 http://dx.doi.org/10.1183/23120541.00377-2020 |
| _version_ | 1783581477905104896 |
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| author | 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 Yiallouros, Panayiotis Zivkovic, Zorica Kuehni, Claudia E. Lucas, Jane S. |
| author_facet | 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 Yiallouros, Panayiotis Zivkovic, Zorica Kuehni, Claudia E. Lucas, Jane S. |
| author_sort | Crowley, Suzanne |
| collection | PubMed |
| description | 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 |
| format | Online Article Text |
| id | pubmed-7487359 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2020 |
| publisher | European Respiratory Society |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-74873592020-09-21 Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia 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 Yiallouros, Panayiotis Zivkovic, Zorica Kuehni, Claudia E. Lucas, Jane S. ERJ Open Res Editorials 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 European Respiratory Society 2020-09-14 /pmc/articles/PMC7487359/ /pubmed/32964005 http://dx.doi.org/10.1183/23120541.00377-2020 Text en Copyright ©ERS 2020 http://creativecommons.org/licenses/by-nc/4.0/This article is open access and distributed under the terms of the Creative Commons Attribution Non-Commercial Licence 4.0. |
| spellingShingle | Editorials 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 Yiallouros, Panayiotis Zivkovic, Zorica Kuehni, Claudia E. Lucas, Jane S. Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title | Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title_full | Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title_fullStr | Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title_full_unstemmed | Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title_short | Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| title_sort | access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia |
| topic | Editorials |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7487359/ https://www.ncbi.nlm.nih.gov/pubmed/32964005 http://dx.doi.org/10.1183/23120541.00377-2020 |
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