Mitochondria-Targeted Exosome Drug Delivery for Methylmalonic Acidemia Neuroprotection An engineered exosome delivery vehicle loaded with antioxidant or enzyme-replacement cargo, surface-functionalized to cross the blood-brain barrier and deposit payload directly into neuronal mitochondria, targeting the root cause of MMAemia-associated brain injury.
Why it helps: Patient advocacy organization that funds research and connects families; a neuroprotective exosome therapy directly addresses their top unmet need — preventing irreversible brain damage in children despite metabolic control.