WESTON, Mass. — Biogen Idec and Isis Pharmaceuticals will work together on a drug invented by Isis for spinal muscular atrophy, the two companies said.
According to the companies, SMA is the most common genetic cause of infant mortality and causes muscle atrophy and weakness, occurring in 1-in-10,000 births. While children with the condition appear normal at birth, symptoms can develop within a few months and significantly shorten their lifespan. The drug, ISIS-SMNRx, is designed to compensate for the underlying genetic defect that causes SMA.