Zolgensma addresses the genetic root cause of SMA by replacing the missing or defective SMN1 gene.
The gene therapy drug “offers children an opportunity to maximize their developmental potential from this one-time therapy,” Hugh McMillan, MD, a pediatric oncologist at Children’s Hospital of Eastern Ontario in Ottawa, said in a Dec. 16 news release. “The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy.”
Zolgensma was developed by AveXis, a biotechnology company acquired by Novartis for $8.7 billion in 2018.
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