Opinion: Curing rare childhood diseases will falter unless Congress steps up
When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter Maggie, then just 6, received the same diagnosis, that answer hadn’t changed.
We soon learned that our situation is tragically common. As many as 30 million Americans live with some type of rare disease, defined as a disease that affects fewer than 200,000 people. About two in three of whom are children. In approximately 95% of cases, the rare disease in question lacks any approved treatment, much less a cure.
