Tag: Spinal muscular atrophy

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 a reported list price of up to $2.6 million in 2025, Zolgensma therapy is one of the most expensive single-dose treatments in the world. This article explains how much does Zolgensma therapy cost for patients and the healthcare system, exploring the complex factors that influence the final price and outlining available financial assistance.

With a list price of over $2 million per dose, Zolgensma is one of the most expensive medications in the world. This staggering cost raises a critical question for families affected by spinal muscular atrophy (SMA): who pays for Zolgensma? The answer involves a complex combination of health insurance coverage, manufacturer assistance programs, and innovative payment models.

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.

In 2019, the FDA's approval of the gene therapy Zolgensma sent shockwaves through the healthcare world with a $2.125 million price tag per dose. This made the treatment for spinal muscular atrophy famously known as 'what is the 2 million dollar pill,' sparking widespread debate about the cost of revolutionary medicine.

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.