Unfortunately I cannot help, as I have no experience with this. It is a very sobering thing to consider, and one my family is not altogether unfamiliar with. I had an uncle that was diagnosed with a very rare form of cancer, and through a very unlikely but not unwelcome turn of events involving a kind nurse and flagging health insurance, his life was saved at the St. Jude Research hospital (in TN).
About twenty years later, he was grown up and was soon to have a family, when the son of a family friend was diagnosed in infancy with the same form of cancer - except it was even more rare. This strain is almost always manifested below the neck (like my uncle), but in this child, it was in his brain. It has been a battle, but the boy was able to fight through it several times. Multiple resurgences later, he is finally recovered: thanks to the research done on my uncle.
All that to say, research on rare diseases is extremely important and something I am very passionate about.
Glad to hear a good story. Sobering is a very good way to put it. For some of the parents, it just brings something else out of them. You lose the option to be a couch potato, forever.
About twenty years later, he was grown up and was soon to have a family, when the son of a family friend was diagnosed in infancy with the same form of cancer - except it was even more rare. This strain is almost always manifested below the neck (like my uncle), but in this child, it was in his brain. It has been a battle, but the boy was able to fight through it several times. Multiple resurgences later, he is finally recovered: thanks to the research done on my uncle.
All that to say, research on rare diseases is extremely important and something I am very passionate about.