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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Publicado en:Orphanet J Rare Dis
Main Authors: 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: Artigo
Idioma:Inglês
Publicado: BioMed Central 2015
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Position Statement
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4417237/
https://ncbi.nlm.nih.gov/pubmed/25902795
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0258-1
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