What It Is Like to Have a Rare Disease. How Clinical Trials Changed My Life?

When I was about 1, I started walking with a stiff gait and stumbling quite a bit. Soon after my parents noticed that I stopped meeting childhood milestones. Worried, they took me to the doctor to learn more. A month before my younger brother was born, my parents discovered why I struggled: I had SMA and it was ultimately a fatal condition.

At the time, there were no real treatments and little research being done on SMA. My parents felt devastated and hopeless. But they turned their fear into action and helped create the SMA Foundation to fund research and encourage better treatments.