At Cure SMA, the safety and well-being of everyone impacted by spinal muscular atrophy (SMA) guide everything we do. We are aware of recent reports of tumors in some people who have been treated with Zolgensma (onasemnogene abeparvovec). We know this news may be unsettling, especially for parents and individuals who have already received Zolgensma or are weighing treatment options right now. We are taking these reports seriously, and we are committed to sharing clear, accurate information with our community as soon as it becomes available.
Novartis has informed healthcare professionals of 9 reports worldwide of tumors diagnosed in children who had previously received Zolgensma. Information about these children is still limited. So far, testing has been completed on the tumors of 3 of the 9 children: 2 were found to be related to Zolgensma treatment, and 1 appears to be unrelated. Regulatory authorities are reviewing these reports, and further information is expected. More than 6,000 children worldwide have received Zolgensma.
Cure SMA has reached out to Novartis, and we are reviewing available safety information with clinical and scientific experts while remaining in contact with regulators and our clinical partners. We will be gathering and sharing more information on what this may mean for children who have already been treated and whether any additional monitoring is recommended.
If you or your child has received Zolgensma and you have questions or concerns, please talk with your SMA care team or treating physician. They know your individual history and are best placed to guide decisions about care and monitoring at this time. Please do not stop or change any part of your care plan without first speaking with your healthcare provider.
Gene therapy has changed what is possible for many people living with SMA, and we have seen its impact firsthand across our community. Like every treatment, it continues to be studied over time, and careful safety monitoring is an essential part of that process. Honest, open communication is how the trust between individuals, families, clinicians, researchers, and industry is built and kept.
We also want to hear from you. If you have questions you want answered, please share them with us at [email protected]. We will share updates here and on our social channels as we learn more.
Cure SMA Support
For questions or support, contact Cure SMA at [email protected] or 800-886-1762.
Media Contact:
Jesse Aynes, CFRE
Sr. Vice President, Development & Marketing
(847) 709-6350
[email protected]
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease affecting motor nerve cells in the spinal cord and the muscles used for breathing, eating, crawling, and walking. SMA affects approximately one in 15,000 births in the U.S., and about one in every 50 Americans is a genetic carrier.
About Cure SMA
Cure SMA is the leading nonprofit organization driving research, advocacy, and support for individuals and families living with SMA. Its mission is to advance research for treatments and a cure, and to support and empower everyone impacted by SMA today.