Since 2017 NDF has been conducting the necessary preclinical studies required to prepare for clinical trials (testing on humans). NDF has committed significant resources toward this potential cure.
Gene therapy is a long process with many steps that can fix faulty genes. It is a treatment that can only be administered to a patient once in a lifetime. Therefore, it is crucial to make sure that scientists know as much as possible before dosing a patient, to ensure safety and efficacy. In order to comply with FDA approval process, many preclinical studies must be completed on blood, muscle tissue, cell-lines and animal models wherever possible, before they can be applied to humans in a clinical trial.
NDF is in the process of completing these studies in order to give gene therapy and other potential, curative treatments the best chance at being effective.