Arnold Gammaitoni, PharmD, Vice President of Medical and Scientific Affairs, Zogenix, discusses survey data demonstrating benefits of fenfluramine for the treatment of Dravet syndrome from caregivers’ perspectives. This data was presented at the AAN 2021 meeting.
Dravet syndrome is a rare neurological condition that usually appears during the first year of life as frequent febrile seizures. As the condition progresses, other types of seizures typically occur, including myoclonus and status epilepticus. Moderate to severe cognitive impairment is also common. Most cases of Dravet syndrome occur due to a mutation of the SCN1A gene. It can be inherited in an autosomal dominant pattern, but most people with Dravet syndrome do not have a family history of the condition
Fenfluramine was approved last year for the treatment of seizures associated with Dravet syndrome. The objective of the study presented at AAN 2021 was to determine how treatment with fenfluramine affected Dravet patients and their families. Participants were parents of children with Dravet syndrome who took part in one-on-one semi-structured interviews to discuss the benefits of the treatment. The most common seizure-related benefits included: reduced frequency of seizures, fewer seizure triggers, shorter recovery when seizures did occur, and improved function following a seizure. The most common non-seizure-benefits included improvements in: cognition, alertness, education, problem-solving, speech, sleep quality, motor function, and mood. Overall, the results of this study suggest fenfluramine is not only effective as a treatment for seizures related to Dravet syndrome but has a positive impact on the quality of life for Dravet patients and their families.
To learn more about Dravet syndrome and other rare neurological disorders, visit checkrare.com/diseases/neurology