(News Bulletin 247) – Novartis has presented new data that underscores the transformational and lasting benefit of Zolgensma ® (onasemnogene abeparvovec), an essential single-use gene therapy for the treatment of spinal muscular atrophy (SMA).

Zolgensma demonstrates sustained durability up to 7.5 years post-dose and 100% achievement of all assessed milestones in children treated prior to onset of SMA symptoms.

‘The latest data from two long-term follow-up studies (LTFU), LT-001 and LT-002, show continued efficacy and durability of Zolgensma in a range of patient populations, with an overall benefit-risk profile that remain supportive,’ the band said.

‘These children now have an improved quality of life, very different from what one would expect if they had not received treatment. I am thrilled to see the new possibilities opening up for children, their families and others who can now receive this treatment,” said Dr. Jerry R. Mendell of Nationwide Children’s Hospital.

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