Spinal Muscular Atrophy affects infants ability to walk, eat, and breath by reducing strength in motor nerve cells in the spinal cord. It is caused by defects in the SMN1 gene. About 400 babies are born with this genetic disorder every year, and ultimately kills each infant in just a few years. Zolgensma, is a drug developed by AveXis is designed to treat Spinal Muscular Atrophy.
Recently approved by the FDA, Zolgensma costs a whopping $2.125 million for a single dose. However, those babies who were administered the drug before 6 months demonstrated less severe muscle problems than those who did not. In fact, the earlier the treatment was administered, the healthier the babies would turn out.
Zolgensma deliveres a healthy copy of the SMN1 gene via adenoassociated virus 9. The genetically engineered virus contains the double-stranded DNA SMN1 transgene and supplements the bodies genetic instruction to produce the SMN proteins. This newly introduced DNA continues to cause protein production and continuous and sustainable rates.