Many Infants With Genetic Epilepsies May Qualify for New Therapies
⚠️ Infant dosing/safety: medication and diet decisions for infants require individualized medical guidance.
Source: medRxiv
Summary
Researchers studied the eligibility of infants with genetic epilepsies for a new type of treatment called antisense oligonucleotide therapy. They looked at 160 infants enrolled in the Gene-STEPS study, which involved genetic testing to identify the causes of their epilepsy. The study took place at four pediatric centers and collected clinical data until October 2025.
The key finding was that about 15.6% of the infants had specific genetic changes that could make them eligible for this new therapy. After considering other factors related to their health and individual characteristics, 64% of those infants could be treated with these therapies now, while an additional 20% could have been eligible at the time their seizures started. This suggests that a significant number of infants might benefit from these advanced treatments.
This research is important because it shows that new therapies could help improve outcomes for infants with genetic epilepsy, who often have limited treatment options. However, the study has some limitations, such as the need for further research to confirm these findings and the fact that not all infants may respond to the therapies. Overall, it highlights the potential for better treatment options in the future.
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