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

Heslop, E and Csimma, C and Straub, V and McCall, J and Nagaraju, K and Wagner, K R and Caizergues, D and Korinthenberg, R and Flanigan, K M and Kaufmann, P and McNeil, E and Mendell, J and Hesterlee, S and Wells, D J (2015) The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. ORPHANET JOURNAL OF RARE DISEASES, 10 (49).

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