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Normally, SMN protein in the body is only produced by the SMN1 gene, but that gene is absent in people with SMA.
Isis Pharmaceuticals Inc., in collaboration with Genzyme Corp., Cold Spring Harbor Laboratory and Harvard Medical School, is advancing a preclinical antisense therapy for the disorder based on the same principle of "fixing" the SMN2 "backup gene." Preclinical trials of that therapy in mouse models showed increased blood levels of SMN protein lasting for up to two weeks, improved survival and function of muscle, and improved survival of motor neurons.The drug is a small, orally bioavailable compound that is designed to up-regulate levels of SMN by targeting the RNA splicing of the SMN2 gene.SMA is caused by mutations in the survival of motor neuron-1 gene (SMN1).In mouse models, PTC observed a 10-fold increase in life span of severely affected animals, which typically live only 14 days."When we heard about this particular program and PTC's approach to it, clearly the science was compelling," Shafique Virani, global head of CNS partnering for Roche, told .