Succinic Semi-Aldehyde Dehydrogenase (SSADH) Deficiency

Succinic Semi-Aldehyde Dehydrogenase (SSADH) Deficiency is a neurological disease caused by genetic mutations that result in elevated levels of succinic semi-aldehyde, a toxic aldehyde that is converted into other metabolites that cause severe neurological dysfunction, including cognitive delay, seizures, and difficulties with strength and movement.  Over 400 patients with SSADH deficiency have been identified worldwide, but there is currently no FDA-approved therapy for SSADH Deficiency. By trapping succinic semi-aldehyde, ADX-102 or other aldehyde traps have the potential to reduce the direct toxicity of succinic semi-aldehyde as well as the formation of neurotoxic metabolites, and represent a novel approach with considerable therapeutic potential in a disease where there remains significant unmet medical need.