New treatment for common genetic disease

It’s an inherited condition that may lie dormant during childhood, but then can cause serious problems with mobility. Charcot  Marie Tooth Disease or CMT is named after the three doctors from England and France who first described it over 100 years ago. It’s a genetic condition that causes problems with the sensory and motor nerves. For some, the progression is so severe they lose their ability to walk.

Twenty-one-year-old Dakota Reilly has had problems with her feet for almost half of her life. 

 "I couldn’t run anymore, and I did have difficulty walking, but I had the mentality that I'm just going to do whatever I can until I physically can’t do it anymore," she said.
 
Dakota was diagnosed with Charcot Marie Tooth Disease; a condition that often leads to weakened muscles on the bottom of the feet. 

"When they’re walking their foot is hanging down and they’re unable to lift it," explained
Dr. Wayne Berberian, an orthopedic surgeon.

As Dakota’s condition worsened, four surgeries over eight months allowed her to walk without braces and start college.

Her mother Kerin Reilly serves on the board of the Hereditary Neuropathy Foundation, and is closely following testing of a new drug for CMT, known now as PX-3003.

"If approved by the FDA,  it’s said to stop the progression and possibly give back a small percentage of what was lost in terms of muscle wasteness," Kerin said.

PX-3003 is a combination of three drugs that are already approved for unrelated conditions. In earlier trials, patients showed significant improvement. Dakota was not in the trial because she has a less common form of CMT, but Kerin says she  hopes her daughter will benefit from the drug, once it is approved. 

The drug is in phase three clinical trials right now, the last stage before the FDA considers it for approval.
 


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