When a $2 million gene therapy falls short: Exploring the limitations and consequences

Patients with spinal muscular atrophy (SMA) who received the gene therapy Zolgensma are increasingly requiring additional drugs, raising questions about the long-term effectiveness and value of high-cost gene therapies. While Zolgensma has shown positive results for many patients, a significant number have needed supplementary treatments to achieve desired outcomes. This challenges the perception that gene therapies can provide a complete cure and raises concerns about the justification of their exorbitant prices. The experience with Zolgensma highlights the need for further research and real-world data to determine the true value and cost-effectiveness of gene therapies.