Thursday, July 25, 2019

FDA Approves Zolgensma As Viable Treatment For Spinal Muscular Atrophy

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.


  1. Its amazing that they have discovered a cure for a such horrible disease that takes a baby away from its parents in the first few years of life. The cost of the cure is insane, leaving those who cant afford it to watch their kid die. I hope that they will find new and better ways to lower the cost of this treatment to make sure that no one has to go through this disease. No one should have to go into debt for the rest of their lives to keep their child alive.

  2. I have heard of Spinal muscular atrophy before, but this is my first time actually reading about it. After doing some research online, i have learned how terrible this disease. With these technological advances it comforts me to know that we have cures for disorders like these. When reading articles like this, it makes me think that science is something from a fantasy! Injecting genetically engineered viruses into babies to alter DNA... Interesting stuff !