New York, Nov. 24, 2025 (GLOBE NEWSWIRE) — The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed by Novartis, a major step forward for families living with spinal muscular atrophy (SMA). The newly approved therapy is indicated for children two years and older, teens, and adults with a confirmed mutation in the SMN1 gene, making it the first and only gene replacement therapy available to this broader SMA population. Itvisma is an intrathecal formulation of Zolgensma — a gene therapy previously approved for infants with SMA — enabling gene replacement therapy to be delivered safely and effectively to older children, teens, and adults for the first time. This expanded treatment option provides renewed hope to people and families living with SMA. Read the Novartis press release here.
The Muscular Dystrophy Association celebrates this important advancement for the spinal muscular atrophy (SMA) community we serve,” said Angela Lek, PhD, Chief Research Officer, Muscular Dystrophy Association. “The FDA approval of Itvisma reflects decades of foundational SMA research and the extraordinary efforts of scientists, clinicians, industry partners and advocates working together to push the field forward. MDA is proud to have supported many of the discoveries that laid the groundwork for today’s milestone. This progress reinforces what is possible when collaboration, innovation, and the voices of families living with neuromuscular disease come together to drive meaningful change.”
“This is a pivotal moment for the SMA community and for the field of neuromuscular medicine,” said Barry J. Byrne, MD, PhD, Chief Medical Advisor and Board Member, Muscular Dystrophy Association, and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. “The approval of Itvisma expands therapeutic options for patients we care for throughout the MDA Care Center Network, who previously had limited access to gene therapy. It reflects the tremendous progress made through years of dedicated research and the growing promise of precision genetic treatments for neuromuscular disease.”
MDA’s Acceleration of Treatments for Spinal Muscular Atrophy
For nearly 75 years, MDA has been the nation’s largest nonprofit supporter of research across more than 300 neuromuscular diseases, including SMA. Through the generous support of donors and partners, MDA has helped fund the foundational science that made gene therapy breakthroughs like Itvisma (onasemnogene abeparvovec-brve) possible. MDA currently supports six active SMA research grants and has invested more than $2 million in SMA research from 2020–2025. Since its inception, MDA has committed nearly $51 million to SMA research, fueling critical discoveries that have reshaped the treatment landscape.
“As someone living with spinal muscular atrophy, I’ve witnessed firsthand the incredible progress our community has made thanks to continued research and innovation,” said Mindy Henderson, Vice President of Disability Outreach & Empowerment at the Muscular Dystrophy Association, who lives with SMA. “This approval represents not only scientific advancement but renewed hope for people and families living with SMA. It inspires a lot of hope in the SMA community to see how far we’ve come, and to know that organizations like the Muscular Dystrophy Association, together with our partners in science and industry, are helping to make what once felt impossible, possible.”
About Spinal Muscular Atrophy (SMA)
SMA is a rare, genetic neuromuscular disease caused by the loss of motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is one of the leading genetic causes of infant mortality, but recent therapeutic advances — including today’s FDA approval — have dramatically improved outcomes and quality of life for affected individuals.
Support and Guidance for Families
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 [email protected].
Media inquiries contact [email protected].
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About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA’s mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, Bluesky, TikTok, LinkedIn, and YouTube.
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years of legacy, impact, and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. Learn more at MDA75.org.
- Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community