Jonah has Sanfilippo Syndrome Type C, a disease that doctors say will eventually kill him. We have been racing the clock ever since, hoping to prove them wrong.
Jonah is eleven years old and appears perfectly healthy and happy. But since he was almost two, his father and I have known that he has Sanfilippo Syndrome Type C, a disease that doctors say will eventually kill him. We have been racing the clock ever since, hoping to prove them wrong. We started a biotech company, funded research for a treatment, and searched for other patients to populate clinical trials. Sanfilippo is ultra-rare: only 19 other individuals in the United States are known to have the disease. Establishing a Rare Disease Center of Excellence at the FDA would go a long way to address the barriers inherent in developing treatments for small patient populations, and that would profoundly advance our efforts.
