Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

Rare or orphan diseases often are inherited and overwhelmingly affect children. Many of these diseases have no treatments, are incurable, and have a devastating impact on patients and their families. Regulatory standards for drug approval for rare diseases must ensure that patients receive safe and...

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Detalles Bibliográficos
Autores principales: Mulberg, Andrew E., Bucci-Rechtweg, Christina, Giuliano, Joseph, Jacoby, David, Johnson, Franklin K., Liu, Qing, Marsden, Deborah, McGoohan, Scott, Nelson, Robert, Patel, Nita, Romero, Klaus, Sinha, Vikram, Sitaraman, Sheela, Spaltro, John, Kessler, Vivian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Position Statement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6368795/
https://www.ncbi.nlm.nih.gov/pubmed/30736861
http://dx.doi.org/10.1186/s13023-019-1017-5

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