Neurology Central

New drug may limit progression of genetic form of ALS

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Results of a preliminary study have demonstrated that a new drug may limit the progression of a genetic form of ALS. The study’s findings were presented at the American Academy of Neurology’s 71st Annual Meeting (PA, USA 4–10 May 2019).

A tenth of all cases of ALS are genetic and of these, a fifth are caused by a mutation in the gene superoxide dismutase 1 (SOD1). “The resulting mutated protein is toxic and leads to ALS by damaging the nerve cells that control movement. Our research aimed to decrease the production of that protein,” explained author of the study, Timothy Miller (Washington University School of Medicine in St. Louis, MO, USA).

“The treatment that we researched in this study, an antisense oligonucleotide called tofersen (BIIB067), works by targeting and reducing protein created by the mutated gene,” continued Miller.

The study involved 50 individuals with ALS

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