Jun 11 2026<\/p>\n
An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.<\/p>\n
![](\"https:\/\/www.europesays.com\/ie\/wp-content\/uploads\/2026\/06\/ImageForNews_839888_17811790838879513.jpg\" "\\"First-in-the-world")
\nFrom right to left: Dr. Dror Kraus, Senior Physician, Neurology Unit and Epilepsy Specialist, Clalit-Schneider Children\u2019s Medical Center; Dr. Naama Ornstein, Head of the Genetics Unit, Clalit-Schneider Children\u2019s Medical Center; Prof Rami I. Aqeilan. Image Credit: Clalit Health Services – Schneider Children\u2019s Spokesperson\u2019s Office<\/p>\n
The treatment was performed at Clalit-Schneider Children\u2019s Medical Center, part of Clalit Health Services, Israel’s largest health organization running 14 hospitals and 1600 community clinics nationwide. It took part after an extraordinary international effort involving physicians, researchers, regulators and biotech leaders who moved urgently to make the impossible possible for one small child.<\/p>\n
The breakthrough began with Dr. Naama Ornstein, head of genetics unit at Clalit- Schneider, who immediately recognized the rare disorder after the baby was hospitalized with seizures and severe developmental decline. Years earlier, she had treated another child with the same condition, but at the time, the experimental therapy had not yet been available, and the child died. Alongside the genetic team, Dr. Dror Kraus, senior physician in the Neurology unit at Clalit-Schneider Children\u2019s Medical Center and an epilepsy specialist, has been closely monitoring and treating the child since the day he arrived at the hospital.<\/p>\n
Determined not to lose another patient, Dr. Ornstein reached out to the scientist who originally developed the therapy, Professor Rami I. Aqeilan, an Arab researcher from the Hebrew University of Jerusalem, whose academic discovery had since evolved into a clinical treatment acquired by a U.S. biotech company led by an Israeli-born physician and CEO.<\/p>\n
Together, the unlikely team crossed borders, systems and bureaucracies to secure special emergency approval for the treatment, despite the fact that it had never before been administered to a human being.<\/p>\n