NEURAL REGENERATION RESEARCH : The evaluation of personalized treatments in vitro of fibroblasts and neuronal cells derived from PKAN patients with a wide range of pharmacological options currently available, and monitoring its effect on the pathophysiological changes, can help for a better therapeutic strategy. In addition, these cell models will be also useful for testing the efficacy of new therapeutic options developed in the future.
This is the same methodology applied to PLAN and, recently, to BPAN and MPAN in the context of BRAINCURE Project.