Novartis experimental drug shows promise in improving motor function for spinal muscular atrophy patients

From Investing.com: 2024-12-30 05:11:34

Novartis AG’s experimental drug shows promise in improving motor function in children with type 2 spinal muscular atrophy. The new treatment, sharing active ingredient with Zolgensma gene therapy, targets older children who can sit unaided but never walked independently. Novartis shares remain stable in Zurich trading, with a 4.4% gain over the past year.

Zolgensma, priced at $2.1 million in the US, was the first million-dollar medicine for babies with spinal muscular atrophy. Novartis plans to present detailed trial results next year and engage with regulators. The high cost of these therapies is justified by their potential to save lives in rare diseases.

The Swiss pharmaceutical company’s new drug will be administered differently from Zolgensma and specifically targets older children with spinal muscular atrophy. Novartis plans to share detailed trial results next year and engage with regulators. The high cost of these life-saving therapies is justified by their potential impact on rare diseases.



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