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

Emma Heslop, Cristina Csimma, Volker Straub, John McCall, Kanneboyina Nagaraju, Kathryn R. Wagner, Didier Caizergues, Rudolf Korinthenberg, Kevin M. Flanigan, Petra Kaufmann, Elizabeth McNeil, Jerry Mendell, Sharon Hesterlee, Dominic J. Wells, Kate Bushby

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Medicine & Life Sciences