RE: [Myotubular_Myopathy] PTC124
Sorry for the error in the previous mail – it is PTC 124. I have no idea if MTM is as a result of a “nonsense mutation”
PTC124 may have the potential to treat many genetic disorders in which a nonsense mutation is the basis for the disease. We are currently conducting initial patient studies in young adults with cystic fibrosis and in children with Duchenne muscular dystrophy. If these clinical trials yield positive results we hope to eventually expand development to multiple genetic disorders. In the meantime, we are continuing our preclinical research in order to assess the potential clinical utility of PTC124 in other genetic diseases.
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