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An Official American Thoracic Society Workshop Report: Translational Research in Rare Respiratory Diseases.

Authors :
Kristof, Arnold S.
Petrof, Basil J.
Hamid, Qutayba
Kolb, Martin
Landry, Jennifer S.
MacKenzie, Alex
McCormack, Francis X.
Murawski, Inga J.
Moss, Joel
Rauch, Frank
Rosas, Ivan O.
Shapiro, Adam J.
Smith, Benjamin M.
Thomas, David Y.
Trapnell, Bruce C.
Young, Lisa R.
Zariwala, Maimoona A.
ATS Assembly on Respiratory Cell and Molecular Biology
Source :
Annals of the American Thoracic Society; Aug2017, Vol. 14 Issue 8, p1239-1247, 9p
Publication Year :
2017

Abstract

Rare respiratory diseases (RRDs) are a heterogeneous group of disorders that collectively represent a significant health care burden. In recent years, strong advocacy and policy initiatives have led to advances in the implementation of research and clinical care for rare diseases. The development of specialized centers and research networks has facilitated support for affected individuals as well as emerging programs in basic, translational, and clinical research. In selected RRDs, subsequent gains in knowledge have informed the development of targeted therapies and effective diagnostic tests, but many gaps persist. There was therefore a desire to identify the elements contributing to an effective translational research program in RRDs. To this end, a workshop was convened in October 2015 with a focus on the implementation of effective transnational research networks and collaborations aimed at developing novel diagnostic and therapeutic tools. Key elements included an emphasis on molecular pathogenesis, the continuing engagement of patient advocacy groups and policy makers, the effective use of preclinical models in the translational research pipeline, and the detailed phenotyping of patient cohorts. During the course of the workshop, current logistical and knowledge gaps were identified, and new solutions or opportunities were highlighted. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
23296933
Volume :
14
Issue :
8
Database :
Complementary Index
Journal :
Annals of the American Thoracic Society
Publication Type :
Academic Journal
Accession number :
124501191
Full Text :
https://doi.org/10.1513/AnnalsATS.201705-406WS