A new treatment for Charcot-Marie-Tooth disease

NEW YORK CITY -- (11/07/2017) -- 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 become wheelchair-bound. Now there’s more on a new treatment that may halt CMT in its tracks.

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,” Reilly told Ivanhoe.

Dakota was diagnosed with Charcot-Marie-Tooth disease; a condition that often leads to weakened muscles on the bottom of the feet.

Wayne S. Berberian, MD, Chief of Foot and Ankle Service, Department of Orthopaedic Surgery at Hackensack University Medical Center explained, “When they’re walking their foot is hanging down and they’re unable to lift it.”

As Dakota’s condition worsened, four surgeries over eight months allowed her to walk without braces and start college at the New York Fashion Institute of Technology.

Dakota’s mother, Kerin Reilly said, “I’m proud of her. She’s thriving and she’s doing extremely well. I mean she has so much struggle every day and she just keeps going.”

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 PXT-3003.

Kerin continued, “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.”

PXT-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.

“It would change her life,” Kerin explained.

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



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