Elpida Therapeutics

Elpida’s mission aims to address the current and significant unmet medical needs of patients with ultra-rare diseases.

Elpida has partnered with CMTA and is conducting a Natural History Study in Charcot-Marie-Tooth Disease Type 4J. The aim of this study is to help clinical researchers gain a better understanding of the disease course in effected individuals that have not been well characterized in the clinic. The study will also help identify more people for clinical trials, inform the design of future clinical trials and identify therapeutic targets, potential biomarkers and endpoints to track treatment effects to be used in a planned gene therapy trial.