AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)!

“In the START clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type 1. ...”
— Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy at The Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, OH
“We are grateful to the tenacious researchers, partners and families who participated in the Zolgensma clinical trials that helped us achieve this incredible milestone,” said Dave Lennon, president of AveXis. “We are proud to bring this one-time gene therapy to pediatric patients with SMA and remain committed to advancing the science behind Zolgensma to transform SMA, as well as other rare genetic diseases.”
— Dave Lennon, President of AveXis

Savsu is really proud to have been selected to help protect this pioneering, life saving, gene therapy with our evo system.

Read announcement HERE