Recent advances in pathophysiological and genetic mechanisms of some 
neuromuscular diseases and a rapid progress in new pharmacological technologies 
led to an accelerated development of innovative treatments, generating an 
unexpected therapeutic revolution. In part 1, we report already commercially 
available drugs, just approved drugs and new therapeutic promises in the 
treatment of peripheral neuropathies. Hereditary transthyretin amyloidosis 
(hATTR) is a devastating disease due to amyloid accumulation in peripheral 
nerves, heart and autonomic system. The first specific drug approved for hATTR 
was tafamidis, a TTR tetramer stabilizer. In 2018, the positive results of two 
phase 3 trials have been reported leading to start of regulatory approval route 
for inotersen, an antisense oligonucleotide and patisiran, the first-ever RNA 
interference (RNAi) therapeutic. System biology targeting approach has indicated 
baclofen, naltrexone and sorbitol in combination (PXT3003) as candidate drugs 
for Charcot-Marie-Tooth disease type 1A. This hypothesis was confirmed in 
experimental models and in phase 2 and 3 clinical trials. Givosiran, another 
RNAi therapeutic, targeting 5-aminolevulinic acid synthase, has been positively 
tested in acute intermittent porphyria in phase 1/2 and ongoing phase 3 trials. 
Although allogenic hematopoietic stem cell transplantation resulted recently a 
long-term therapy in mitochondrial neurogastrointestinal encephalomyopathy 
(MNGIE), a new strategy is liver transplantation which is able to revert the 
severe biochemical and clinical imbalance of the disease. Recently, a gene 
therapy has been tested in a MNGIE murine model, indicating that it may become a 
new therapeutic option.