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The therapy reduces expression of a gene called SOD1, which in some cases of familial amyotrophic lateral sclerosis (ALS) has a mutation that weakens and kills nerve cells called motor neurons that control muscle movement.
Mutations in the FUS/ TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis.
Other studies presented suggest that NAALADase inhibitors can delay the appearance of muscle weakness in a transgenic mouse model of familial amyotrophic lateral sclerosis and are potent non-narcotic analgesics in several animal models of chronic pain.