From the Research
Newer Treatments for Lafora Disease
- Gene replacement therapy has shown promise as a potential treatment for Lafora disease, with studies demonstrating its ability to ameliorate neurological and histopathological alterations, reduce epileptic activity and neuronal hyperexcitability, and decrease the formation of Lafora bodies 1, 2.
- Gene therapy using a recombinant adeno-associated virus carrying the human EPM2A gene has been shown to improve symptoms in mouse models of Lafora disease, with benefits lasting up to nine months after a single injection 1, 2.
- Dapagliflozin, a SGLT2 transporter inhibitor, has been proposed as a potential treatment for Lafora disease, with a study showing its ability to ameliorate the disease phenotype in a zebrafish model by reducing neuronal hyperexcitability, locomotor impairment, and spontaneous seizure-like events 3.
- Current treatments for Lafora disease are limited to antiepileptic drugs, which are often ineffective in the long term, highlighting the need for new and effective therapies 4, 5.
Potential Therapeutic Strategies
- Gene therapy and gene replacement therapy are being explored as potential treatments for Lafora disease, with several studies demonstrating their promise in mouse and zebrafish models 1, 2, 3.
- Repurposing of existing drugs, such as dapagliflozin, may also offer a potential therapeutic strategy for Lafora disease 3.
- Early and accurate diagnosis of Lafora disease is crucial for the development and implementation of effective treatments, and for improving patient outcomes 5.