These Patients Got the Cure. Then It Went Away.

The article highlights the troubling trend of gene therapies for rare diseases being developed but ultimately discarded by drug companies due to the high costs of production for larger patient populations. This situation is concerning as it not only affects the availability of potentially life-saving treatments but also raises questions about the sustainability of innovation in the pharmaceutical industry. The implications for patients who could benefit from these therapies are significant, as they may be left without viable options.