The White Coat Cape: An Ethical Analysis of Emerging Therapies to Treat Spinal Muscular Atrophy

The recent emergence of promising therapies to treat neuromuscular diseases such as spinal muscular atrophy raises important questions regarding the ethical permissibility of allowing a parent to refuse these Food and Drug Administration-approved drugs. The 3 most recent drugs targeting spinal muscular atrophy have all been approved since 2019, lack long-term data regarding potential side-effects and long-term benefits, and are costly. Indeed, onasemnogene abeparvovec-xioi (Zolgensma) has been called the most expensive drug in the world. Contemporary analyses of innovative therapies, compassionate use medications, off-label usage, and emerging therapies tend to focus on the importance of informed consent in framing the ethical dimensions of these medications. This manuscript utilizes a narrative framework of “rescue” to explore the competing perspectives of optimistic physicians and parents, who may decline the therapies finding the benefit-burden profile does not weigh in favor of their use. Ultimately, this paper concludes that such refusal should be considered ethically permissible until such time as more long-term data are available for these medications and their cost has decreased substantially.