Novartis(US:NVS) Globenewswireยท2025-11-25 03:21Core Insights - The FDA's approval of Itvisma (onasemnogene abeparvovec-brve) is a significant advancement for families affected by spinal muscular atrophy (SMA), marking the first gene replacement therapy available for a broader SMA population, including children aged two and older, teens, and adults [1][2] - Itvisma is an intrathecal formulation of Zolgensma, previously approved for infants, allowing safe and effective delivery of gene therapy to older patients for the first time [1][2] - The Muscular Dystrophy Association (MDA) has played a crucial role in supporting foundational research that led to this breakthrough, having invested nearly $51 million in SMA research since its inception [3] Company and Industry Overview - The MDA has been the largest nonprofit supporter of research for over 300 neuromuscular diseases, including SMA, for nearly 75 years, demonstrating a long-standing commitment to advancing treatment options [3][9] - The approval of Itvisma reflects the collaborative efforts of scientists, clinicians, and industry partners, showcasing the potential of precision genetic treatments for neuromuscular diseases [2][4] - SMA is a rare genetic neuromuscular disease that leads to progressive muscle weakness and is one of the leading genetic causes of infant mortality, but recent advancements have significantly improved patient outcomes and quality of life [5]