Gene Therapy in the Era of "Incurable": The Promise and Price of Nusinersen for SMA

Abstract

The advent of gene therapy offers unprecedented hope for treating genetic disorders like Spinal Muscular Atrophy (SMA), yet its transformative potential is often undermined by prohibitive costs. This paper focuses on Nusinersen, an effective therapy that modifies the SMN2 gene to produce life-sustaining proteins, significantly altering the prognosis for SMA patients. Despite its clinical success in restoring motor functions and stabilizing disease, Nusinersen’s exorbitant price tag transforms a medical breakthrough into a financial battleground, restricting access primarily to those with substantial resources or government support. By contrasting the barriers to care with the positive model of public funding in Hong Kong, this work contends that the right to advanced medical treatment should be universal. It concludes that genetic therapies like Nusinersen must be recognized as essential healthcare, guaranteed to all patients regardless of their economic standing.