Treatment with metformin in twelve patients with Lafora disease.

Francesca BisulliLorenzo MuccioliGiuseppe d'OrsiLaura CanafogliaElena FreriLaura LicchettaBarbara MostacciPatrizia RiguzziFederica PondrelliCarlo AvolioTommaso MartinoRoberto MichelucciPaolo Tinuper

Published in: Orphanet journal of rare diseases (2019)

Metformin was overall safe in our small cohort of LD patients. Even though the clinical outcome was poor, this may be related to the advanced stage of disease in our cases and we cannot exclude a role of metformin in slowing down LD progression. Therefore, on the grounds of the preclinical data, we believe that treatment with metformin may be attempted as early as possible in the course of LD.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
stem cells
prognostic factors
combination therapy
mesenchymal stem cells
big data
artificial intelligence
smoking cessation