In this episode of Let’s Talk About That, Anirvan Ghosh speaks with Arya Singh, a rare disease advocate, researcher, and policy leader whose life story intersects with one of the most remarkable transformations in modern genetic medicine.

Arya was diagnosed at 18 months with spinal muscular atrophy (SMA)—a devastating neuromuscular disease that, until recently, was the leading genetic cause of death in infants. At the time, there were no treatments and little hope. What followed was a race against time: Arya’s parents helped launch the SMA Foundation, building the scientific collaborations that ultimately enabled the development of multiple life-changing therapies.

Arya herself became one of the earliest participants in the clinical trials that led to those breakthroughs—receiving the first antisense therapy for SMA and later transitioning to an oral treatment that transformed her strength, stamina, and independence.

In this conversation, Arya reflects on growing up inside a revolution in medicine, the courage required to participate in experimental trials as a child, and how scientific progress reshaped her future.

Now pursuing a PhD in population health sciences at Weill Cornell, Arya is working to ensure that the lessons of SMA—about science, advocacy, and policy—continue to drive progress for patients with rare diseases around the world.