The Muscular Dystrophy Association (MDA)  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. 

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.”