TACT Reviews

A non-confidential summary of the TACT report is detailed below however interested parties and potential funders are encouraged to contact the researcher directly to request a copy of the full TACT report.

October
2011


HT-100 - Halofuginone

DMD

Marc B Blaustein MPP
Halo Therapeutics LLC

A randomised, double-blind, placebo-controlled, multiple-dose, dose-escalation study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of HT-100 in patients with Duchenne muscular dystrophy





Naproxcinod - HCT 3012

BMD

Fabrizio Dolfi
NicOx SA

A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.





Human Embryonic Stem Cell Derived Motor Neuron Progenitors

SMA

Chris N Airriess
California Stem Cell

Human Embryonic Stem Cell Derived Motor Neuron Progenitors for the Treatment of Motor Neuron Disease.



January
2011


Tetracycline Derivatives

SMA

Dr Higgins
Paratek Pharmaceuticals

Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA. Screen of a tetracycline library -identified a group of potential candidates. One of these will be selected as a final candidate.





GsMTx4

DMD

Dr Sachs
Rose Pharmaceuticals

Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels.



June
2010


N-Acetyl cysteine

CMD

Dr Ferreiro
Institute of Myology

Oxigem: AntiOXIdants for a GEnetic Myopathy. The First Clinical Trial for a Congenital Muscle Disorder: N-Acetyl cysteine (NAC) Treatment for SEPN1 Related Myopathy.





P-188

DMD

Dr Symons
Phrixus Pharmaceuticals

An open-label, fixed dose, exploratory study to assess the efficacy and safety of P-188 NF on left ventricular volume changes in patients with Duchenne muscular dystrophy (DMD)





Laminin-111

DMD

Dr Hodges
Prothelia Inc

Recombinant human Laminin-111 for treatment of Duchenne Muscular Dystrophy



February
2010


lisinopril / Losartan

DMD

Dr Chris Spurney
Children's National
Medical Center

Treatment of early cardiac systolic dysfunction in Duchenne muscular dystrophy with lisinopril or Losartan: a prospective, randomized, blinded, crossover trial.






Isosorbide dinitrate plus Ibuprofen

DMD

Professor Clementi &
Dr D'Angelo
L Sacco University Hospital

A pharmacological treatment for muscular dystrophy combining NO-releasing and non steroidal anti-inflammatory drugs.





Flavocoxid

DMD

Professor Vita &
Dr Messina
University of Messina

Randomized double-blind placebo-controlled trial of flavocoxid in Duchenne muscular dystrophy.
 
05 Jan 2012