Experimental Treatments for Charcot–Marie–Tooth Disease

ADX71441 is an experimental therapy with the potential to slow the progression of nerve damage in people who have CMT disease. The compound appears to have only been tested in preclinical studies so far, although it was approved for in-human trials in 2013.

Mexiletine is being investigated for use as a muscle relaxant in patients with CMT disease. A completed Phase 2 trial showed that mexiletine has the potential to reduce the duration and intensity of muscle cramps in CMT patients.

Modafinil is a medication that promotes wakefulness and was approved to treat sleepiness in some sleep disorders. Although modafinil has not been approved specifically for the treatment of CMT, research has shown that it can reduce fatigue in this group of patients.

PTX3003 is being developed to reduce the levels of PMP22 gene expression. It is a combination of three already approved treatments: baclofen, naltrexone, and sorbitol. A pivotal Phase 3 trial is currently enrolling.

Ricolinostat (ACY-1215), designed to restore nerve function by inhibiting the histone deacetylase 6 protein, is being developed to treat CMT type 2, as well as diabetic and chemotherapy-induced peripheral neuropathy. A Phase 2 trial in CMT2 patients is currently enrolling.