PXT 3003 na Doença de Charcot Marie Tooth.

PXT3003 is a combination of baclofen, naltrexone, and D-sorbitol developed to address the primary cause of CMT1A: the overexpression of the PMP22 protein. In preclinical studies, the medication reduced PMP22 expression, improved myelination, and preserved neuromuscular function. In clinical trials, the highest dose demonstrated a significant functional benefit compared to placebo, although of modest magnitude. Despite promising results, subsequent studies have not definitively confirmed its efficacy, and the drug has not yet been approved for clinical use. Even so, PXT3003 marked an important advance by demonstrating that it is possible to develop therapies targeting the molecular mechanisms of CMT1A. #CMT #CMT1A #CharcotMarieTooth #PXT3003 #PMP22 #HereditaryNeuropathy #NeuromuscularDisease #Neurology