(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.
Copyright (c) 2023 News Bulletin 247. All rights reserved.
I have over 8 years of experience working in the news industry. I have worked as a reporter, editor, and now managing editor at 247 News Agency. I am responsible for the day-to-day operations of the news website and overseeing all of the content that is published. I also write a column for the website, covering mostly market news.