Magazine Article | February 7, 2018

When Treatment For A Rare Disease Is On The Horizon, Hope Rises — And So Does Doubt

Source: Life Science Leader

By Joy Aldrich

“There is nothing I can do for you. There is no treatment or cure.”

That’s what a neurologist told us when he diagnosed my mother, brother, and me with Charcot-Marie-Tooth (CMT), a hereditary neuromuscular disease that would progressively get worse as we got older. I was 14 at the time, and my mom was already having trouble getting up and down stairs and with daily tasks such as using a knife, buttoning a shirt, and turning a key to open a door.