Cure Rare Disease™ is developing custom therapeutics that are as unique to the individuals they are meant to treat. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors. Our customized therapeutics are designed specifically for the men and women who continue to fight for their right to live long, full, healthy lives despite having been diagnosed with a rare genetic disorder for which they’ve been told there is no treatment or cure.
THE STORY OF CURE RARE DISEASE
I used to hold my breath every time I saw it was my mom calling.
I’m not sure if that pit in your stomach feeling is “normal” for others or not, I just know that whenever I was away from home, there was very little I could do to keep it at bay.
My family and I are close.
We know that when push comes to shove we’re all each other have got, and that means we take care of each other—no matter what.
Growing up, when something went wrong at my parent’s convenience store, we were there until late sorting it out. Later when they decided to expand and open a carwash, we knew it would be up to us to figure out any issues the machines might have, fix them up and get things back in working order. The time those machines spent offline was money that wasn’t coming through the door putting my family at risk.
It was a big responsibility, but we knew that as long as we tackled our problems together nothing could hold us back.
My family has each other’s backs. We figure things out, and we always keep moving forward.
It was no different when it came to my brother Terry. My parents, grandparents, and great-grandparents were all too familiar with Duchenne muscular dystrophy. By the time Terry was diagnosed, they’d already watched as their sons, brothers, and uncles lost their mobility—and eventually their lives—to this rare disease.
Time was ticking away for my brother, but back then, all I knew was that my brother was sick and that we traveled pretty regularly to see doctors. A lot of doctors.
The older I got the more I understood what “sick” actually meant and the sense of urgency my parents had around getting my brother seen by as many doctors and specialists as they could.
For Terry, sick didn’t mean a runny nose, antibiotics, or missing school here and there.
It meant walking into a movie theater only to get stuck at the steps leading to our seats because he could no longer lift his leg on his own because of a progressive disease.
The panicked look that washed across both my father and brother’s faces that day is something I will never forget, even after 15 years.
It was one of those moments when things went from being okay to terribly not okay within seconds.
Kind of like the day my mom called and said: “Terry’s fallen again.”
Falls weren’t uncommon for my brother but with every fall the chances of him not being able to walk again increased dramatically.
I was far from home and felt completely helpless. My family needed me, but I found myself having just started business school and months away from being able to be home with them.
Terry’s time, independence, and mobility were continuing to slip away with no ability to fight back. The feeling of hopelessness became a never-ending theme in my life and the lives of my family.
As I repeatedly hit send, I held my breath—just like when my mom would call. Except this time, it wasn’t fear I was feeling… this time it was determination. I was hopeful that my school’s name would be enough to get the attention and help of even one of the people I’d reached out to.
And it was.
What happened over the next 12 months was a blur–an organization was established with an unprecedented collaboration of the world’s leading researchers passionately embarking on a journey to stop a disease that has never had a survivor.