Journal article

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



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 limited numbers of patients who can be enrolled into clinical trials. TREAT-NMD Advisory Committee for Therapeutics (TACT) was established to provide independent and objective guidance on the preclinical and development pathway of potential therapies (whether novel or repurposed) for NMD.We present our experience in the establishment and operation of the TACT. TACT provides a unique resource of recogn..

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Awarded by TREAT-NMD from the European Commission through FP6

Awarded by Second Public Health Programme

Awarded by Medical Research Council

Awarded by US department of defence

Awarded by National Institute for Health Research

Funding Acknowledgements

We acknowledge funding support for TREAT-NMD from the European Commission through FP6 (contract number 036825) and the Second Public Health Programme (contract number 2012 3307), as well as support from the Medical Research Council (grant number MR/K501074/1), US department of defence (Award W81XWH-11-1-0782 K Nagaraju, CNMC, USA), Parent Project Muscular Dystrophy and Cure Duchenne.Support from patient organisations - Parent Project Muscular Dystrophy (PPMD), Cure Duchenne, Association Francaise contre les Myopathies (AFM), Jesses Journey, Muscular Dystrophy Campaign (MDC), Muscular Dystrophy Association (MDA), Action Duchenne, Cure CMD, and Foundation to Eradicate Duchenne (FED).