NTF3 and Charcot-Marie-Tooth disease, axonal, type 2FF: Moreover, a gene therapy involving intramuscular injection of an AAV1 encoding human neurotrophin-3 under the control of a muscle-specific promoter, which was successfully tested in a mouse model of Charcot Marie Tooth neuropathy (Sahenk et al., 2014), now has FDA approval for a clinical trial for patients with Charcot Marie Tooth 1A (Professor Zarife Sahenk, personal communication, August 2016).