Encouraging early study for investigational drug in people with motor neurone disease – University of Sheffield News

Results from Phase 1-2 of a study published in the New England Journal of Medicine, have shown the safety and tolerability of the new investigational drug tofersen which shows the potential to reduce the levels of a toxic cell protein in people living with a form of motor neurone disease (MND); triggered by a faulty gene called SOD1 which causes the progression of the disease.

SOD1 is the known cause for triggering MND in two per cent of all patients with ALS, and up to 20 per cent of patients who have a family history of the disease.

Click here to read full article https://www.sheffield.ac.uk/news/nr/new-investigational-drug-for-mnd-patients-1.893013


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