My 13-year-old daughter Eliza suffers from a rare, fatal disease known as Sanfilippo Syndrome. The neurodegenerative illness causes children to gradually lose their mental abilities starting around age three. Other symptoms include seizures, vision and hearing impairment, severe sleep disturbance, and loss of speech and physical abilities. Children diagnosed with the disease typically die in their teens.
Worst of all, despite what appears to be a major medical breakthrough on treatments for Sanfilippo, the Food and Drug Administration has refused to funnel the medicine through a faster approval process that is the only hope these children have.
They and their advocates aren't asking for some exceptional policy change at the FDA – just recognition that the emerging Sanfilippo treatments are exactly what the agency's "accelerated approval" path is designed to expedite.
