Imagine giving birth to your first child and having the doctor tell you that they have a rare disease and will probably never see their fifth birthday. Or taking your toddler to numerous specialists because they aren’t developing quite like your other children, only to find out they will never learn to walk and talk. Or overcoming the shock of a child born deaf only to be told that they will begin going blind before they can even drive a car.
Everyday, about 72 children around the world are born with a rare disease. That’s 72 babies who face a life of struggle and pain, 72 parents who will live in fear and heartache, and 72 families that will face a world turned upside down, draining their time, energy, and money.
Now, image that there is a way to stop the effects of a rare disease by treating patients with proven science, BUT it’s just not cost-effective to create this treatment. There is not enough money to be made bringing lifesaving, life-altering therapies to patients simply because there aren’t enough people with the disease. This is the world that thousands and thousands of families live in today.
A child is born with a rare disease, and the doctor says, “Don’t worry. It will be alright. We have a treatment that will mitigate the effects of this disease and your child will live a happy and full life.”
Which world would you like to live in?