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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

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