This is a wonderful film that can be leveraged in so many ways to help the Rare Disease community fight for change.
Learn about the “Too Rare To Care” Team by reading their mission below:
Rare diseases affect more people than Cancer and AIDS combined.
There are over 8,000 rare diseases in the world. 1 in 10 people worldwide have a rare disease and 50% of these rare diseases affect children. But unfortunately, there are treatments for less than 5% of those diseases. As such, 30% of these children don’t make it to their 5th birthday. And while no one thinks their child will be born with a rare disease, it can happen to anyone – nature does not discriminate.
We can appreciate the battle families of kids with rare diseases fight. The mission of our film is to raise awareness about the diagnostic and cure odyssey of rare disease kids that falls primarily on the advocacy of parents. We ask the question, why isn’t more being done to save these kids? And if this was your child, what would you do?
When parents are told that their child has a rare genetic disease, they are advised that nothing can be done, that there are no treatments, and to “go home and enjoy your child.” But the science and success of gene and cell therapy is changing the landscape for rare diseases and giving families hope.
But finding a therapy and treating one child with a rare disease can cost up to 6 million dollars. And most pharmaceutical and Biotech companies are not incentivized because there is little to no profit margin developing drugs for rare diseases. So families must raise millions of dollars on their own and find scientists and Biotech companies willing to help save their child
We need to change the way we think about, talk about, research and cure rare diseases. We need to think of them in the aggregate, just like we think about over 200 diseases that we call simply call “cancer.” The limits to research funding, the challenges to therapy development, the resistance to commercialization, in other words, our failure to save these children from these diseases stems from one characteristic – that they are rare.