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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: COPD Foundation, Inc 2015
Assuntos:
Conference Report – Alpha-1 Foundation
Acesso em linha: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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