Table 1.
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 |
For full details see http://www.treat-nmd.eu/resources/tact/reviews/past/).