• by George Simpson , Featured Contributor, September 21, 2017

There is a little-known condition called Charcot-Marie-Tooth Disease that affects one in 2,500 people (about the same prevalence as multiple sclerosis) including 150,000 Americans and nearly 3 million people around the world. It is often not correctly diagnosed because it is relatively uncommon.

For example, Christina Olson, the subject of Andrew Wyeth's iconic painting "Christina's World," was first thought to have polio. Now, most doctors agree she was dragging herself across the field because she was crippled by CMT.

CMT can vary greatly in severity, even within the same family. It can cause severe disability -- and, in rare instances, even death. It is slowly progressive, causing deterioration of peripheral nerves that control sensory information and muscle function of the foot/
lower leg and hand/forearm, leading to significant problems with movement, touch, and balance as it advances. It can be -- and often is -- passed from parent to child.

Roy Behlke, a former designer with Pratt & Whitney Aircraft, always had trouble with foot drop and his arches. As a young man, he joined the Boy Scouts, but the mandatory 10-mile hikes were exhausting for him, since they frequently required crossing creeks on logs and homemade bridges. Roy ended up falling into creeks on more than one occasion. In later years, as his condition worsened, Roy saw an orthopedic doctor, who diagnosed him with arthritis and said he would need to have all his major joints replaced, one by one. Alarmed at the diagnosis, Roy saw a rheumatologist, who pointed him to a neurologist and finally a correct diagnosis of CMT at the age of 55.

It's estimated that tens of thousands of people have CMT, but don't yet know it. Steve Snow passed CMT to his daughter without realizing. Five years later, she died. A female neurologist in Washington failed to recognize her husband's symptoms until they discovered their child had CMT.

Until now, there has been no cure for CMT. One reason is that diseases that affect many more people are better bets for pharma companies because they create a larger market for new drugs. Everyone is touched by cancer in some way; not so many are exposed to the effects of CMT.

More progress on a potential treatment/cure has been made in the past year than at any time since CMT was identified in 1886. Studies in two rodent models of CMT1A not only stopped the progression of the disease, but also showed improvement in some symptoms. Now, funds are urgently needed to prepare for clinical trials. But it will cost $15 million to bring to market.

I hope you will join me in helping to jump-start funding for this vital research by clicking here. I thank you, my daughter, who has CMT, thanks you -- as do all those who suffer from this devastating condition, and those who work on its eventual eradication.