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✔ Disease Type/Condition: Charcot-Marie-Tooth (CMT)

Charcot-Marie-Tooth (CMT)

PXT3003 in Charcot-Marie-Tooth Type 1A

The purpose of this study is to evaluate the effectiveness of an investigational drug called PXT3003 for the treatment of Charcot-Marie-Tooth type 1A (CMT1A disease). Researchers will compare PXT3003 to placebo (inactive substance) on the ...