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Table 1 Previous applications (Feb 2010 – October 2014)

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

Name Country Disease Drug Stage of Development Industry/Academia
Christopher F. Spurney, MD Children’s National Medical Center USA DMD Losartan/lisinopril Clinical Academia
Emilio Clementi MD, PhD & Grazia D’Angelo, MD, PhD H Sacco University Hospital Italy DMD Isosorbide dinitrate plus ibuprofen Clinical Academia
Giuseppe Vita, MD & Sonia Messina MD, PhD University of Messina Italy DMD Flavocoxid Clinical Academia
Ana Ferreiro, MD Institute of Myology France CMD N-Acetylcysteine Clinical Academia
James Symons, M.S., PhD Phrixus Pharmaceuticals Inc USA DMD P-188 Clinical Industry
Bradley L Hodges, PhD Prothelia Inc USA DMD Laminin-111 Preclinical Industry
Paul Higgins, PhD Paratek Pharmaceuticals USA SMA Screen of a tetracycline library one to be selected as a final candidate Preclinical Industry
Fredrick Sachs, PhD Rose Pharmaceuticals USA DMD GsMTx4 Preclinical Industry
Mark Blaustein Halo Therapeutics USA DMD Halofuginone (HT-100) Clinical Industry
Fabrizio Dolfi, MD, PhD NicOx SA France BMD Naproxcinod (HCT 3012) Clinical Industry
Chris N Airriess, PhD California Stem Cell USA SMA Human Embryonic Stem Cell Derived Motor Neuron Progenitors Clinical Industry
Fred Marin, PhD GMP-Orphan SAS France SMA Satisma (sodium phenylbutyrate) Preclinical Industry
Urs Ruegg, PhD University of Geneva Switzerland DMD Tamoxifen Preclinical Academia
Joel Braunstein, MD, FACC, MBA Tivorsan USA DMD Biglycan Preclinical Industry
Paolo Bettica, MD, PhD Italfarmaco Italy DMD Givinostat Clinical Industry
Dariusz C Gorecki MD, PhD University of Portsmouth UK DMD P2X7 Preclinical Academia
Joanne Donovan, MD, PhD Catabasis Pharma, Inc. USA DMD CAT-1004 Clinical Industry
Erica Reeves, PhD ReveraGen BioPharma, Inc. USA DMD VBP15 Preclinical Industry
Patricio Sepulveda Myostin Therapeutics Pty Ltd Australia DMD Myostin Preclinical Industry
Peter Flynn, PhD Fate Therapeutics Inc. USA DMD Wnt7a Preclinical Industry
Jon Tisley, PhD Summit PLC UK DMD SMTC 1100 Clinical Industry
Jens Schmidt, PhD University Medical Centre, Göttingen Germany IBM DMF/BG12 & 1400 W Preclinical Academia
Denis Guttridge, PhD Ohio State University USA DMD NBD Therapy Preclinical Academia
Carl Morris, PhD Pfizer Inc. USA DMD Anti-GDF-8 Ab Clinical Industry
Mimoun Azzouz, PhD University of Sheffield UK SMA Viral vector to deliver the SMN transgene Preclinical Academia
Richard Franklin, PhD Tarix Orphan LLC USA DMD TXA127 Preclinical Industry
Seth Porter, PhD FibroGen USA DMD FG-3019 Clinical Industry
Ennio Ongini, PhD & Gloria Vigliani, MD TrophyNOD France DMD Naproxcinod Clinical Industry
Suyash Prasad, MD Audentes Therapeutics USA XLMTM AAV8 Clinical Industry
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