How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Among the challenges confronting patients with rare diseases is a dearth of treatment options. The development of safe and effective new therapies is hampered by challenges associated with conducting clinical trials in small populations. In this article, we describe how the Duchenne muscular dystrop...

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Detalles Bibliográficos
Autores principales: Furlong, Pat, Bridges, John F. P., Charnas, Lawrence, Fallon, Justin R., Fischer, Ryan, Flanigan, Kevin M., Franson, Timothy R., Gulati, Neera, McDonald, Craig, Peay, Holly, Sweeney, H. Lee
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4486430/
https://www.ncbi.nlm.nih.gov/pubmed/26104810
http://dx.doi.org/10.1186/s13023-015-0281-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4486430/
https://www.ncbi.nlm.nih.gov/pubmed/26104810
http://dx.doi.org/10.1186/s13023-015-0281-2

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