Eight-year-old Leonor struggles to walk, sit up straight and hold a glass of water. Other kids make fun of her because she has to wear leg braces. When Leonor was diagnosed with CMT, doctors said she’d ben in a wheelchair for life. Her family refuses to accept that fate, and so do we. There’s always something we can do. Learn how you can take action to end CMT.

By: Gary Donaldson The New York Times recently published an article about individuals living with rare genetic diseases who have become advocates for their conditions. These courageous champions are shaping research to change the course of their disease, despite the...

By Patrick Livney, Chairman of the Board Recently, I was contacted by Barton, a son of Charles G. Bentzin,  who informed me of the passing of his father. Sadness aside, his reaching out touched me on two fronts. First, telling me how Charles “enjoyed talking with you...