It's estimated that 25 million Americans have diseases that are very rare. As our National Correspondent Jon Frankel has discovered, drug companies are often reluctant to develop cures for rare diseases because there's so little chance to make a profit.
Fighting a fatal disease is one of the toughest challenges a family can face. For one Indiana family, it's a crisis compounded by an unhelpful system.
Phil and Tricia Milto have been married for eight years. These days they don't have much time for each other. Phil works in his family dry-cleaning business, but together, the Milto's full-time occupation is fighting to save their six-year-old son's life.
It all began two years ago when their son, Nathan went in for what was supposed to be a routine check up. "We had tests done. The doctor comes back and says he has this Batten disease. I looked at the doctor and said, 'is that bad?'" recalls Nathan's father, Phil.
It is worse than they could have ever imagined. Batten disease attacks the nervous system. Because it strikes only about 3 in one hundred thousand children--little is known about it. The Milto's started looking for answers as Nathan began losing his sight. Now he's blind. Unless a therapy is discovered, Nathan is not expected to live past age twelve.
Nathan's dad faces another struggle: navigating the world of medicine. With a charity called Nathan's Battle, Phil is attempting to bring researchers and doctors together and convince them that it is financially feasible to find a cure for his son "...to try and solve this rare disease using a gene therapy," he says. "Then hopefully expand that to other diseases and so then hopefully that would expand the bottom line for these companies."
Nathan's passion is sports. He loves his hometown team--the Indiana Pacers. "He doesn't really understand what's happening to himself. So he just doesn't ask any questions, like 'why could I see one day now I can't. Why could I play basketball before and now I can't,'" says his mother, Tricia.
When Nathan plays basketball, dad bangs his wedding band on the basket to guide him. "I just give him the ball and I use my wedding ring to tap on the rim," says Phil. "He hears it and he goes for that sound. He shoots close to that noise and sometimes he actually makes it."
Nathan's younger brothers could also have Batten disease. A simple blood test holds the answer. But Tricia is reluctant to have them tested. "Then everyday you live wondering if this is the day they show their first symptom," says Tricia.
Tricia and Phil both carry the gene for the disease. So their kids have a one in four chance of getting it. "Our time, our days are numbered," says Phil. "We're just trying to change that and extend it as long as we can,"
The Milto's hope a new gene therapy could save Nathan. But clinical trials are at least a year and a half away. In the meantime, Phil continues to raise money and attention for Batten Disease reearch.
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