Why is a CMT4J natural history study important?

The purpose of a Natural History Study of Charcot-Marie-Tooth 4J (CMT4J) is to better understand CMT4J. It aims to:

  • Understand and document the medical history, clinical characteristics, progression of symptoms, and medical events in individuals with CMT4J.
  • Provide greater understanding of CMT4J by determining outcome measures or a desired solution that a potential therapy could provide for use in future clinical trials.