Mechanisms and therapeutic targeting of motor neuron death in SMA

Breakthrough SMN-­inducing therapies have recently been approved by FDA for the treatment of SMA. However, they do not provide a cure and therapeutic approaches that address unmet needs of SMA patients by enhancing the clinical benefit of SMN-­inducing therapies through combinatorial treatment are highly sought after. To address this issue, our project aims to elucidate the mechanisms underlying the initiation and execution of motor neuron degeneration in SMA as well as establish the therapeutic potential of SMN-independent, pharmacological targeting of this pathway for neuroprotection.