One-of-a-Kind drug created for single Patient's unique genetic disease

This study is testing a custom-made drug designed specifically for one person with a rare, inherited nerve disorder called Charcot-Marie-Tooth disease type 2D. The drug is an antisense oligonucleotide, which is a type of molecule that aims to target the underlying genetic cause of the disease. Researchers will monitor the single participant for two years to see if the treatment is safe and if it helps improve hand and arm function and quality of life.