Publication
Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA
E.P. Hoffman, Carsten Bonnemann, Marc Boutin, Bernard Brais, Filippo Buccella, Arthur Burghes, Christopher Coffey, Nabarun Dasgupta, Hugh Dawkins, Annamaria De Luca, Christopher Dowd, Tina Duong, Michelle Eagle, Richard Finkel, Pat Furlong, Cynthia Gagnon, Nathalie Goemans, Michela Guglieri, Yetrib Hathout, Nicholas Johnson, Emil Kakkis, Petra Kaufmann, Jonathan Kimmelman, Lawrence Korngut, Joyce Kullman, Hanns Lochmüller, Stefano Marini, Craig McDonald, Charles Mohan, Lauren Morgenroth, Hiroki Morizono, Kanneboyina Nagaraju, John Porter, Lori Reilly, Markus Rüegg, Joel Schneider, Pietro Spitali, Volker Straub, Lee Sweeney, Giorgio Tasca, Cathy Turner, Olav Veldhuizen, Jan Verschuuren, Susan Ward, Raffaella Willmann
Neuromuscular Disorders, July 2017, Elsevier
DOI: 10.1016/j.nmd.2017.02.013