The Cure SMA Foundation congratulates the FDA on the broad approval of Itvisma for the treatment of

The Cure SMA Foundation, a global leading non-profit organization dedicated to spinal muscular atrophy (SMA), announced today its welcome for the U.S. Food and Drug Administration (FDA) approval of Novartis' Itvisma™ (onasemnogene abeparvovec-brve) for the treatment of patients aged 2 years and older with all types of SMA. This one-time gene therapy delivers a functional SMN1 gene to motor neurons, helping restore SMN protein expression and potentially slowing or halting disease progression. Clinical data demonstrate that Itvisma improves motor function and produces rapid therapeutic effects, expanding treatment options for older children and adult patients.

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"This approval is a long-awaited milestone for the SMA community," said Kenneth Hobby, President of the Cure SMA Foundation. "Families of adult patients and older children have been eagerly awaiting this treatment. Since receiving community funding in the early basic research phase, our core goal has been to develop novel gene delivery technologies that can benefit more older patients. By preserving motor function, this one-time gene therapy creates new possibilities for enhancing patients' independence in daily life."

The Cure SMA Foundation emphasized that this breakthrough is first and foremost attributed to the entire SMA community—patients, families, clinicians, researchers, and advocates who have advanced the research process over decades through participating in clinical trials, policy advocacy, and raising critical funds. Over the past 30 years, the community has invested more than $92 million in research funding, laying the scientific foundation for today's breakthrough.

"While celebrating this milestone, we are also focused on the future," said Dr. Jackie Glasscock, Chief Scientific Officer of the Cure SMA Foundation. "Our immediate priority is to ensure families have access to the educational support needed to understand the therapy, as well as the key resources to obtain treatment. We will continue to engage with payers and healthcare institutions to ensure the community fully benefits from this new option."

About Spinal Muscular Atrophy (SMA)

SMA is a progressive neurodegenerative disease that primarily affects motor nerve cells in the spinal cord, leading to impairments in muscle function such as breathing, eating, crawling, and walking. Approximately 1 in 15,000 newborns in the United States is affected by SMA, and 1 in 50 people is a carrier of the disease-causing gene.

About the Cure SMA Foundation

As a leading non-profit organization, the Cure SMA Foundation is committed to advancing SMA research, policy advocacy, and patient support to accelerate the development of therapies and empower individuals and families affected by SMA.