These Patients Got the Cure. Then It Went Away.

The article discusses the challenges faced by patients with rare diseases who have access to gene therapies, only to have the treatment discontinued by pharmaceutical companies. Gene therapies, while promising, are often developed for small patient populations, making it financially unviable for companies to produce and distribute them on a larger scale. The article highlights the case of patients with spinal muscular atrophy (SMA), a rare genetic disease, who received a groundbreaking gene therapy treatment called Zolgensma. However, the drug's manufacturer, Novartis, later stopped producing the treatment due to financial constraints, leaving patients and their families in a difficult situation. The article also examines the broader issue of the high cost of developing and manufacturing gene therapies, which can make it challenging for pharmaceutical companies to continue producing these treatments for a larger patient population. This problem is particularly acute for rare diseases, where the potential market is small. The article underscores the need for a more sustainable and equitable approach to the development and distribution of gene therapies, ensuring that patients with rare diseases have access to life-changing treatments.