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Statement from the Muscular Dystrophy Association on Reports of Tumors in People Treated with Zolgensma
New York, NY, Thursday, November 5, 2026 – The health, safety, and well-being of people living with spinal muscular atrophy (SMA) and their families are always our highest priority at the Muscular Dystrophy Association (MDA). We are aware of recent reports of tumors in people who have received Zolgensma (onasemnogene abeparvovec) and understand that this raises questions and concerns for families, particularly those who have made important treatment decisions for themselves or their children. We take these reports seriously and believe families deserve transparent, timely, and science-based information as more is learned.
Limited information is available currently about the timing of events, nature of the tumors, and any future monitoring of treated individuals that may become appropriate. MDA is seeking additional information, reviewing the available scientific and safety data with clinical and research experts, and has reached out to Novartis. We encourage families with immediate questions or concerns about treatment history, potential risks, or recommended monitoring to speak with their healthcare providers, who can offer guidance based on individual circumstances.
Gene therapy represents a significant advancement in the treatment of genetic diseases, including SMA, and has brought meaningful progress and new possibilities to many people and families. These advances are helping change the course of diseases that, not long ago, had limited treatment options. At the same time, gene therapy is a complex and evolving field, and understanding both its benefits and potential long-term risks requires continued research, careful monitoring, and open communication.
We recognize the sense of urgency from families to access additional information about these reports in order to make informed treatment and monitoring decisions. MDA is working to learn more as soon as possible. We also would like to recognize the important work of the researchers, clinicians, and companies advancing these treatments and believe continued collaboration and transparency are essential to sustaining progress and maintaining the trust of the communities these therapies are intended to serve.
MDA remains committed to listening to and supporting the SMA community and helping ensure families have access to credible, timely, and science-based information as additional evidence emerges.
MDA Support
For more information on SMA, and ongoing support for families and medical professionals, contact the MDA Resource Center by phone 1-833-ASK-MDA1 (1-833-275-6321) or email ResourceCenter@mdausa.org.
Media contacts:
Muscular Dystrophy Association: press@mdausa.org
Novartis: media.relations@novartis.com
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) has been at the center of progress for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions for over 75 years. We unite researchers, clinicians, advocates, and families to speed the pace of discovery, improve access to expert care, and ensure inclusion in every aspect of life. Our mission is simple: give the people we serve the tools and opportunities to live longer, more independent lives. To learn more visit mda.org. Follow MDA on social media on Instagram, Facebook, X, TikTok, LinkedIn, and YouTube.
