Janet Stewart, MSc,  —

The European Medicines Agency has agreed to Pharnext SA’s pediatric investigation plan for PXT3003, an investigational therapy being developed to treat Charcot-Marie-Tooth disease type 1A (CMT1A). Because CMT1A typically appears in the first 20 years of a person’s life, Pharnext intends to explore the use of PXT3003 as…

Defects in a gene responsible for Charcot-Marie-Tooth type 2B (CMT2B) appear to interfere with a process in cells called autophagy, a mechanism for the elimination of cellular waste. These findings suggest that normalizing autophagy in CMT2B patients may have therapeutic benefits. The study, titled “Alterations of autophagy in the…