Retrophin's investigational agent for Duchenne Muscular Dystrophy (DMD, a degenerative muscle disease). RE-001 is a recombinant fusion protein designed to substitute for dystrophin that is lacking in DMD patients. Human clinical study of RE-001is expected to begin in 2012.
Rupjani: This is the first I'm hearing of this. Not quite sure how it works. Is it the combination of genes to make a working dystrophin protein? Does anyone have anymore information on this?
As per my knowledge it is developed by Dr. James Ervasti at the University of Minnesota. Dr. Ervasti conducted crucial animal studies that demonstrated the potential efficacy of Tat-utrophin as a treatment for Duchenne.
TAT Utrophin is a utrophin molecule combined with another cell penetrating agent so that it can easily penetrate the cell.