Tag: Sma

While Zolgensma has been a life-altering gene therapy for infants with Spinal Muscular Atrophy (SMA), experts widely agree that **Why is Zolgensma not a cure?** is a critical question for managing patient expectations. The single-dose treatment halts disease progression but cannot reverse pre-existing neurological damage, a key distinction from a complete cure.

With an initial price tag exceeding $2.1 million per dose, Zolgensma became known as the world's most expensive medicine upon its approval in 2019. This staggering cost is the result of a complex interplay of factors, including its groundbreaking gene therapy technology, a small patient population, and the innovative pricing strategies employed by pharmaceutical companies.

According to developers at Scholar Rock, despite significant advances in treatments for spinal muscular atrophy (SMA), progressive muscle weakness remains a major unmet medical need. In this context, apitegromab is an investigational muscle-targeted therapy being developed to address the muscle atrophy and weakness that contribute to functional decline in SMA patients.