When my husband Dave and I learned that two of our three daughters had a fatal genetic disease, we entered the kind of nightmare for which no parent can fully prepare. Both girls — Keira and Livvy — were diagnosed with metachromatic leukodystrophy, or MLD, a rare degenerative disorder that destroys the nervous system and gradually robs children of the ability to walk, speak, swallow, and ultimately survive.

But Keira had something that Livvy did not: time. 

Recommended Stories

FDA should follow Trump’s lead on medical ‘right to try’

Modern science can reverse heart disease. Washington prefers cheap statins

The FDA’s new leadership must give rare disease patients a clearer path