Novartis: more positive data for Zolgensma

(CercleFinance.com) - Novartis reports further positive data for its Zolgensma, highlighting the safety and efficacy profile of this gene therapy in children with spinal muscular atrophy weighing between 8.
5 and 21 kg, with an average age of 4.69 years.

The final data from the SMART study thus add to a growing body of evidence on the use of Zolgensma in a patient population that is older and heavier (1.5 to 9.1 years) than the children treated in previous clinical studies.

Almost all treated patients maintained or improved their motor milestones after 52 weeks, most of whom had switched to the single gene therapy from another chronically administered disease-modifying therapy, Novartis said.


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