Novartis is in the trial phase for a new experimental medicine that has shown promise in improving motor function in children with a form of spinal muscular atrophy, according to a Dec. 30 report from The Detroit News.
A study of the new treatment, which shares the active ingredient of Novartis' gene therapy Zolgensma, included children with spinal muscular atrophy type 2 who had never received treatment, were aged two or older and could sit but had never walked independently.
Zolgensma was first introduced five years ago and was the first potential cure for babies with spinal muscular atrophy. The new medicine would be administered differently and target older children, according to the report.
When it was first introduced, Zolgensma was priced at $2.1 million in the U.S., making it the first medicine to cost more than $1 million.