UCSF trial offers hope for children with Dravet syndrome, rare and severe childhood epilepsy

"Oli's mom, Lindsay Dagan, described the severity of his condition, stating, 'We were in the hospital every week with seizures. His seizures wouldn't stop on their own, so we'd have to give rescue meds, often multiple doses that still wouldn't stop the seizure.'"

"Dr. Nilika Singhal explained the significance of the experimental drug, saying, 'With this medication, the beauty is that people with Dravet syndrome still have one intact, functioning copy of the gene. And so what happens is that this medication is able to enhance the function of the good copy.'"

"Lindsay shared the impact of the syndrome on Oli's social life, noting, 'So it was really hard for him to have any friends at all, because we had to kind of shelter him from everything.'"

Oli, a ten-year-old with Dravet syndrome, has faced severe seizures that disrupt his life. His family frequently visited hospitals for treatment. Researchers at UCSF are testing new therapies, including cannabis-based medications. Recently, Oli joined a clinical trial for an experimental drug, STK001, which aims to enhance the function of a healthy gene copy. This innovative approach provides new hope for children suffering from this debilitating condition, potentially improving their quality of life and ability to engage with peers.

Read at ABC7 San Francisco