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European Medicines Agency accepts marketing authorization of ALS treatment
2 monthes ago - By Healio
Biogen announced that the European Medicines Agency has accepted the marketing authorization application for tofersen, an investigational drug for the treatment of superoxide dismutase 1 ALS.
According to a company press release, superoxide dismutase 1 ALS is a rare, progressive and fatal disease with no treatment, affecting approximately 2% of people with ALS. The antisense oligonucleotide drug tofersen has been designed to bind to SOD1 mRNA, prompting its degradation and reducing SOD1 protein production.
The marketing authorization application includes results from the phase 3 VALOR
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