MPAN is a rare, progressive neurodegenerative disorder for which there is currently no disease-modifying treatment. Our work is focused on changing that. We are developing a two-stage therapeutic strategy designed to address MPAN at more than one level. Rather than targeting only the consequences of the disease, our approach aims to first tackle the underlying biological defect and then support the recovery and protection of affected cells. Stage 1 — Correct the underlying disease mechanism The first stage focuses on restoring the biological function disrupted in MPAN. By targeting the root molecular cause of the disease, we aim to reduce the cellular dysfunction that drives progressive neurodegeneration. Stage 2 — Protect and restore vulnerable cells Correcting the underlying defect may not be enough once cells have already been under stress for many years. The second stage therefore focuses on helping vulnerable cells recover, improving their resilience and creating the conditions needed for lasting therapeutic benefit.