Crowdfunding for neuromuscular disease treatment: the ethical implications

The arrival of effective gene therapies for congenital neuromuscular disease is enormously encouraging; however, it brings ethical challenges. In the past 5 years, many families of patients with spinal muscular atrophy have resorted to public appeals for financial support (crowdfunding). This has been accelerated by the approval of the single-dose gene therapy onasemnogene abeparvovec, considerered the most expensive drug in the world at about...