20 . Are there any new therapeutic approaches for Dravet syndrome ?

Research in neurosciences and in neurology is very active, bringing hope for the future. Some research topics on new therapeutic approaches are discussed but we currently have no practical answer.

Regarding treatments, it is unclear why the therapeutic response differs from one patient to another. One of the hypotheses suggests that genetic factors would have an influence. This hypothesis needs further investigations.

Regarding new therapeutic treatments, there are more and more discussions about the use of medical marijuana (pills or syrup) to treat seizures. For the time being as we write this booklet, there is currently no clinical evidence for its efficacy in Dravet syndrome. The hope seems to have started with the story of a young girl with Dravet syndrome who experienced an incredible improvement of her condition with the use of a specific strain of marijuana. Even if medical marijuana has been tried in other patients, there is a real need to assess its efficacy and safety before initiating its regular use in children with Dravet syndrome. However, experimental studies in epi­lepsy have already focused on cannabidiol with mixed results making it difficult to draw any conclusion.

Clinical trials could provide a clearer answer in a near future.

Regarding already marketed drugs, some are undergoing clinical trials to assess their efficacy and safety in Dravet syndrome. Your child’s doctor may ask if you are willing to enroll your child into clinical trials, to help the development of possibly new coming treatments.

The use of stem cells in epilepsy is currently also under investigation in research labs only. If they ever reached mass market, they would only treat the symptoms but not cure Dravet syndrome.

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