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Innovation Observatory > Reports > Drugs > PXT3003 for Charcot-Marie-Tooth Disease Type 1A

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PXT3003 for Charcot-Marie-Tooth Disease Type 1A


Neurology and Neurosurgery

January 2018

Charcot-Marie-Tooth disease type 1A (CMT1A) is an inherited genetic disease in which the motor and/or sensory peripheral nerves are affected, resulting in muscle weakness and wasting, as well as sensory loss. These symptoms occur first in the legs and later in the hands. The nerve cells in individuals with this disorder are not able to send electrical signals properly because of abnormalities in the nerve or in the insulation around the nerve. Specific gene mutations are responsible for the abnormal function of the peripheral nerves. Over time, affected individuals may lose the normal use of their feet, hands, legs and arms. Common problems can include decreased sensitivity to heat, touch or pain, muscle weakness in the hand, foot or lower leg, trouble with fine motor skills, loss of muscle mass in the lower leg, and frequent tripping or falling. The disease is slowly progressive and variable, and those affected may have difficulties with every-day activities and may have a shorter life expectancy.

There is currently no cure for CMT1A, and treatments include physiotherapy, splints, occupational therapy and sometimes surgery. PXT3003 is a combination of 3 different ingredients that targets the specific mechanisms involved in the nerve abnormalities in CMT1A. It is formulated as an oral solution. If licensed, PXT3003 would be the first treatment for patients with CMT1A.

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