What is Charcot-Marie-Tooth
Disease (CMT)?

Charcot-Marie-Tooth disease (CMT) is the most common inherited peripheral nerve disorder and is sometimes referred to as hereditary motor sensory neuropathy (HMSN). CMT causes damage to the nerves, which carry signals to and from the brain and spinal cord to the rest of the body. CMT results in muscle weakness and some loss of sensation in the feet, legs, hands and forearms. It can also cause stiffened joints, resulting in high-arched feet and sometimes curvature of the spine (scoliosis). There are many types of CMT, so the exact symptoms can vary from person to person. The A083-03 study is researching two of the most common forms, CMT type 1 and CMT type X (CMT1 and CMTX).

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ACE-083 is an investigational product that is currently being studied and has not been approved by any regulatory agency for any use.