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

1 Newcastle University, Newcastle upon Tyne, UK

2 Cydan Developments Inc, Cambridge, USA

3 Pharma Mac, Boca Grande, USA

4 Children’s National Medical Center, Washington, USA

5 Johns Hopkins University, Baltimore, USA

6 Genethon, Evry, France

7 University of Freiburg, Freiburg, Germany

8 Nationwide Children’s Hospital, Columbus, USA

9 National Institutes of Health, Washington, USA

10 Parent Project Muscular Dystrophy, New Jersey, USA

11 Royal Veterinary College, London, UK