Novartis announced positive results from a trial of its gene therapy for spinal muscular atrophy (SMA), a rare muscle disorder. The therapy, branded as Zolgensma, improved motor function in children aged 2 to less than 18 with SMA. It aims to address the lack of protein necessary for neuromuscular development in patients. The therapy showed improvement in sitting, rolling, crawling, and standing abilities. SMA affects about 1 in 10,000 people and is a leading genetic cause of infant deaths. Other approved treatments for SMA include Spinraza and Evrysdi. Novartis plans to share study results with regulatory agencies next year.
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Gene Therapy Improves Motor Function in Children With SMA
