Arya Singh, SMA patient and accessibility advocate

Some of the most memorable and formative times in Arya Singh’s childhood were spent participating in clinical trials.

Her parents enrolled her in a clinical trial for a treatment for spinal muscular atrophy (SMA), a severe, progressive neuromuscular disease, when she was ten years old. She grappled with some tough experiences in the trial – she missed school, was in and out of the hospital, and didn’t know how to communicate with her peers about what she was going through.

When Arya eventually joined another clinical trial for a small molecule treatment for SMA, she initially approached the situation with fear, but soon she was pleased with her decision to participate. Despite the ups and downs of being part of clinical trials – the challenges, confusion, frustration, as well as gratitude and hope – being part of clinical research was something consistent in her life. Arya reflected on her early years and realized that participating in these trials was a gift. These were experiences that not only helped her but shaped her path into adulthood. They were therefore at the forefront of her mind when she began her college application process.

The thought of going to college was at first something Arya (and her parents) approached with some hesitancy and fear – it was the first time she’d be moving away from home, getting thrown into an entirely new environment. But it had always been a dream of hers, a way to prove to her peers she was on equal footing with them. Despite the challenges ahead, especially knowing that many of the schools she applied to struggled with accessibility, Arya was determined. She was guided by a phrase she and her dad held close: If there’s a will, there’s a way. When she was accepted to Yale and started her freshman year, it became the mantra guiding her experiences as someone with a disability on a college campus.

At first, Arya didn’t want her college experience to be completely defined by SMA. A series of events early on in her freshman year changed that and led her to becoming a vocal advocate for SMA, rare diseases and people with disabilities at Yale and in the New Haven community. She was proud and it felt rewarding to be able to see the conversation about disabilities taking place, and to see change happen all around her as a result.

This all further emphasized for Arya how important it is for the patient’s voice to be a part of the clinical trial process. It drove her to write a children’s book, focused on bringing to life the experience of being a part of clinical trials and helping children as they embark on this unfamiliar journey. As she looks ahead to the future, she wants to work in healthcare in some capacity – working for and with patients to ensure their experiences are reflected in clinical research.

I think there is no other pharma company, especially in the United States, that is this committed and loyal to patients with rare diseases, and I think PTC’s work in SMA is a remarkable example of that.
Arya Singh SMA patient and accessibility advocate

That’s what Arya admires about PTC. It was through clinical trials, and the SMA Foundation – the advocacy organization founded by her parents – that she first became aware of PTC. Their commitment to patients with rare diseases had a strong impact on her, as she felt it spoke volumes to the people and the since behind PTC. Looking back, the collaboration and partnership between PTC and the SMA Foundation to finding a treatment for SMA was a leap of faith, but it was a success. Or, to put it differently: If there’s a will, there’s a way.