PXT3003 is currently in clinical development for treatment of patients aged 16-65 years with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT is a group of inherited conditions that damage the peripheral nerves (reside outside the brain and spinal cord). CMT1 is characterised by muscle weakness and atrophy, which can lead to repeated ankle sprains, and changes in sensation which can cause clumsiness. CMT is a progressive disease meaning that the symptoms get worse over time. Currently, there is no specific treatment approved for CMT1A (symptomatic or disease modifying); current disease management options are limited to supportive care (such as leg braces, physiotherapy, anti-depressant, pain killer, surgery).
PXT3003 for treating Charcot-Marie-Tooth disease type 1A
PXT3003 is currently in clinical development for treatment of patients aged 16-65 years with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT is a group of inherited conditions that damage the peripheral nerves (reside outside the brain and spinal cord).