The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development

Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in rare diseases with large unmet needs and a limi...

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Detalles Bibliográficos
Autores principales: Heslop, Emma, Csimma, Cristina, Straub, Volker, McCall, John, Nagaraju, Kanneboyina, Wagner, Kathryn R, Caizergues, Didier, Korinthenberg, Rudolf, Flanigan, Kevin M, Kaufmann, Petra, McNeil, Elizabeth, Mendell, Jerry, Hesterlee, Sharon, Wells, Dominic J, Bushby, Kate
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Position Statement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4417237/
https://www.ncbi.nlm.nih.gov/pubmed/25902795
http://dx.doi.org/10.1186/s13023-015-0258-1

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4417237/
https://www.ncbi.nlm.nih.gov/pubmed/25902795
http://dx.doi.org/10.1186/s13023-015-0258-1

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