TreatSMA

Zolgensma shows impressive results in older children when administered by intrathecal injection

A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children.

Basel, Switzerland-based drugmaker Novartis said Saturday that interim data from a Phase I/II study of Zolgensma showed an increase by several points on a scale used to evaluate motor function in children aged 2 to less than 5 with untreated SMA Type 2 when administered into an area of the spine. Patients achieved a median increase of 5.9 points from baseline on the Hammersmith functional motor scale-expanded, or HFMSE, compared with a median increase of 4.2 points when data were presented in May at the American Academy of Neurology’s annual meeting.

Please click here to read the full company press release.

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