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Article

Treatment with metformin in twelve patients with Lafora disease

21 Jun 2019

Abstract excerpt

BACKGROUND: Lafora disease (LD) is a rare, lethal, progressive myoclonus epilepsy for which no targeted therapy is currently available. Studies on a mouse model of LD showed a good response to metformin, a drug with a well known neuroprotective effect. For this reason, in 2016, the European Medicines Agency granted orphan designation to metformin for the treatment of LD. However, no clinical data is available...

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PMID: 31227012
DOI: 10.1186/s13023-019-1132-3

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