The parents of twins Amelia and Makenzie Kahn, who have been battling Batten disease for eight years, are observing signs of progress after one year in a clinical study at UNC Health. The personalized treatment, Zebronkysen, led by Dr. Yael Shiloh-Malawsky, is showing promise in managing the disease.
Amelia and Makenzie received their first dose of Zebronkysen in June 2024. The drug was developed by Dr. Michelle Hastings from the University of Michigan for their specific mutation associated with juvenile Batten disease, or CLN3. Since then, improvements have been noted: Makenzie can walk farther unassisted and Amelia has started eating again.
Batten disease is a fatal pediatric neurodegenerative disorder that affects motor skills, communication, cognitive abilities, vision, and more. Patients often face seizures and systemic issues like feeding difficulties. Life expectancy ranges from teenage years to early twenties.
The ForeBatten Foundation funds research into juvenile Batten disease and supports affected families. In June 2024, it launched an “N-of-2” clinical trial at UNC for a rare mutation of CLN3 with Dr. Shiloh-Malawsky leading the study.
Over the past year, Zebronkysen has shown benefits for the girls by helping restore their gene function and preventing further decline. Notably, Makenzie’s energy levels improved significantly as she became less drowsy and more alert.
Karen Kahn observed changes in Amelia as well: “Before the treatment Amelia experienced sensory overload with Batten,” she said. “Now she tolerates noise in a restaurant and is happy.”
Both girls showed temporary regained abilities after each dose without serious adverse events reported related to the study drug.
In June 2025 they received their target high dose of 45 milligrams which will be maintained every three months if benefits continue according to Dr. Shiloh-Malawsky: “Our hypothesis was that restoring CLN3 protein function would stabilize this neurodegenerative disorder…seeing improvements is more than we hoped for.”
Looking ahead toward expanding treatments beyond current mutations affecting other patients globally; scientists hope funding efforts can support development work on medications benefiting larger populations affected by similar conditions.
David Kahn remarked positively on results so far stating “We haven’t had any more loss…that speaks stabilization.”
