Currently, there is no cure for ECHS1 Deficiency. The families of the affected children have a mission to raise 350.000 US Dollars to start preclinical trials for ECHS1 Deficiency at UT Southwestern, Dallas, Texas, where scientists can focus on developing the ECHS1 Deficiency gene therapy.
After the preclinical research another 350.000 USD is needed for the toxicology study to submit the research results for FDA approval.
As of December 2020, a total of 245.000 USD has been transferred by the families to a dedicated account at UT Southwestern Medical Center.
Donate on the official page created by UT Southwestern: Click here