New research offers hope for treating inherited peripheral neuropathies
specifically Charcot Marie Tooth disease types 1A (CMT1A) and 1B (CMT1B). By focusing on increasing levels of cyclic ...
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ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease
In October 2024, ENCell reported the results of a Phase 1 clinical trial investigating the safety and exploratory efficacy of repeated low-dose administration of EN001 in CMT type 1A patients.