Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A

Arthur Suckow, PhD, chief executive officer at DTx Pharma talks about Charcot-Marie-Tooth Disease Type 1A (CMT1A) and a treatment in development for this rare disease.

CMT1A is a rare inherited neurological disorder that causes damage to the peripheral nerves. It is caused by a duplication of the PMP22 gene that leads to poor myelination of axons. Current treatment options are symptomatic and may include physical therapy, occupational therapy, braces and other orthopedic devices, orthopedic surgery, and pain medications.

DTx Pharma is developing a treatment for CMT1A that reduces the duplication of the PMP22 gene. More specifically, the company is developing a method to deliver short interfering RNAs (siRNAs) that inhibit the expression of disease causing genes. The company is finalizing its preclinical studies and plans to begin clinical trials in late 2023 or early 2024.

To learn more about CMT1A, visit checkrare.com/charcot-marie-tooth-disease-type-1a/