Story perspectives

World’s First Gene Therapy Stops Infant Epilepsy Seizures

6/10/2026

Summary

Story summary

An eight-month-old infant received the world’s first experimental gene therapy for severe genetic epilepsy.
The therapy, given at Schneider Children’s Medical Center, delivered a healthy WWOX gene copy to brain neurons in one injection, leaving the infant stable and seizure-free one month later, though long-term follow-up is needed.
Professor Rami Aqeilan expects an FDA application in two months and hopes clinical-trial phases could produce a medicine in 2-3 years.

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Story summary

An eight-month-old infant received the world’s first experimental gene therapy for severe genetic epilepsy.
The therapy, given at Schneider Children’s Medical Center, delivered a healthy WWOX gene copy to brain neurons in one injection, leaving the infant stable and seizure-free one month later, though long-term follow-up is needed.
Professor Rami Aqeilan expects an FDA application in two months and hopes clinical-trial phases could produce a medicine in 2-3 years.