Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia

Suzanne Crowley; Inês Azevedo; Mieke Boon; Andrew Bush; Ernst Eber; Eric Haarman; Bulent Karadag; Karsten Kötz; Margaret Leigh; Antonio Moreno-Galdó; Huda Mussaffi; Kim G Nielsen; Heymut Omran; Jean-François Papon; Petr Pohunek; Kostas Priftis; Bernhard Rindlisbacher; Francesca Santamaria; Arunas Valiulis; Michal Witt; Panayiotis Yiallouros; Zorica Zivkovic; Claudia E Kuehni; Jane S Lucas

doi:10.1183/23120541.00377-2020

Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia

Suzanne Crowley^*, Inês Azevedo, Mieke Boon, Andrew Bush, Ernst Eber, Eric Haarman, Bulent Karadag, Karsten Kötz, Margaret Leigh, Antonio Moreno-Galdó, Huda Mussaffi, Kim G Nielsen, Heymut Omran, Jean-François Papon, Petr Pohunek, Kostas Priftis, Bernhard Rindlisbacher, Francesca Santamaria, Arunas Valiulis, Michal WittPanayiotis Yiallouros, Zorica Zivkovic, Claudia E Kuehni, Jane S Lucas

^*Corresponding author af dette arbejde

Afdeling for Børn og Unge

3 Citationer (Scopus)

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.

Originalsprog	Engelsk
Tidsskrift	ERJ Open Research
Vol/bind	6
Udgave nummer	3
Sider (fra-til)	00377-2020
ISSN	2312-0541
DOI	https://doi.org/10.1183/23120541.00377-2020
Status	Udgivet - jul. 2020

Adgang til dokumentet

10.1183/23120541.00377-2020Licens: CC BY

Citationsformater

Crowley, S., Azevedo, I., Boon, M., Bush, A., Eber, E., Haarman, E., Karadag, B., Kötz, K., Leigh, M., Moreno-Galdó, A., Mussaffi, H., Nielsen, K. G., Omran, H., Papon, J.-F., Pohunek, P., Priftis, K., Rindlisbacher, B., Santamaria, F., Valiulis, A., ... Lucas, J. S. (2020). Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia. ERJ Open Research, 6(3), 00377-2020. https://doi.org/10.1183/23120541.00377-2020

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, J-F, Pohunek, P, Priftis, K, Rindlisbacher, B, Santamaria, F, Valiulis, A, Witt, M, Yiallouros, P, Zivkovic, Z, Kuehni, CE & Lucas, JS 2020, 'Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia', ERJ Open Research, bind 6, nr. 3, s. 00377-2020. https://doi.org/10.1183/23120541.00377-2020

@article{dd7f5ef0ef664a77b00069666de16376,

title = "Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia",

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.",

author = "Suzanne Crowley and In{\^e}s Azevedo and Mieke Boon and Andrew Bush and Ernst Eber and Eric Haarman and Bulent Karadag and Karsten K{\"o}tz and Margaret Leigh and Antonio Moreno-Gald{\'o} and Huda Mussaffi and Nielsen, {Kim G} and Heymut Omran and Jean-Fran{\c c}ois Papon and Petr Pohunek and Kostas Priftis and Bernhard Rindlisbacher and Francesca Santamaria and Arunas Valiulis and Michal Witt and Panayiotis Yiallouros and Zorica Zivkovic and Kuehni, {Claudia E} and Lucas, {Jane S}",

note = "Copyright {\textcopyright}ERS 2020.",

year = "2020",

month = jul,

doi = "10.1183/23120541.00377-2020",

language = "English",

volume = "6",

pages = " 00377--2020",

journal = "ERJ Open Research",

issn = "2312-0541",

publisher = "BMJ Publishing Group Ltd",

number = "3",

}

TY - JOUR

T1 - Access to medicines for rare diseases

T2 - beating the drum for primary ciliary dyskinesia

AU - Crowley, Suzanne

AU - Azevedo, Inês

AU - Boon, Mieke

AU - Bush, Andrew

AU - Eber, Ernst

AU - Haarman, Eric

AU - Karadag, Bulent

AU - Kötz, Karsten

AU - Leigh, Margaret

AU - Moreno-Galdó, Antonio

AU - Mussaffi, Huda

AU - Nielsen, Kim G

AU - Omran, Heymut

AU - Papon, Jean-François

AU - Pohunek, Petr

AU - Priftis, Kostas

AU - Rindlisbacher, Bernhard

AU - Santamaria, Francesca

AU - Valiulis, Arunas

AU - Witt, Michal

AU - Yiallouros, Panayiotis

AU - Zivkovic, Zorica

AU - Kuehni, Claudia E

AU - Lucas, Jane S

PY - 2020/7

Y1 - 2020/7

N2 - 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.

AB - 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.

U2 - 10.1183/23120541.00377-2020

DO - 10.1183/23120541.00377-2020

M3 - Journal article

C2 - 32964005

SN - 2312-0541

VL - 6

SP - 377

EP - 2020

JO - ERJ Open Research

JF - ERJ Open Research

IS - 3

ER -

Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia

Abstract

Adgang til dokumentet

Fingeraftryk

Citationsformater