Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases

Clinical research in rare diseases, including alpha-1 antitrypsin deficiency (AATD), faces challenges not shared by common disease research. These challenges may include the limited number of patient volunteers available for research, lack of natural history studies on which to base many clinical tr...

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Bibliografiske detaljer
Udgivet i:Chronic Obstr Pulm Dis
Main Authors: Wanner, Adam, Groft, Stephen C., Teagarden, J. Russell, Krischer, Jeffrey, Davis, Barry R., Coffey, Christopher S., Hickam, David H., Teckman, Jeffrey, Nelson, David R., McCaleb, Michael L., Loomba, Rohit, Strange, Charlie, Sandhaus, Robert A., Brantly, Mark, Edelman, Jonathan M., Farrugia, Albert
Format: Artigo
Sprog:Inglês
Udgivet: COPD Foundation, Inc 2015
Fag:
Conference Report – Alpha-1 Foundation
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5556970/
https://ncbi.nlm.nih.gov/pubmed/28848840
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15326/jcopdf.2.2.2015.0132
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