Therapeutic Benefits

As with Parkinson’s Disease, research has shown that the gene DYT1 is mutated in primary torsin dystonia and codes for a dysfunctional version of the protein, TorsinA. Preclinical research in the Caldwell laboratories at the University of Alabama has shown that co-expression of the “correct” version of TorsinA decreases the protein clumping associated with the mutant protein. From these results, it is likely that expression of the normal TorsinA gene, or the use of existing drugs that activate the expression of normal TorsinA or inactivate mutant TorsinA, could ameliorate these diseases at a causative level. QRxPharma’s goal is to develop and commercialize existing drugs with a known history of use that will activate the Torsin system and alleviate the cause of Parkinson’s Disease, dystonias and other neurodegenerative diseases.