Among the various subtypes of CMT, CMT1A is the most common form, characterized by a duplication of the PMP22 gene. Despite being a well-known genetic abnormality associated with CMT1A ...
Notably, about 75% of diagnosed cases stem from mutations in two genes: PMP22, which is associated with CMT1A, and MPZ, ...
The study enrolled CMT1A patients, whose disease was caused by a mutation in the PMP22 gene on chromosome 17. The Paris-based company said that the trial was scuppered by a high placebo response ...