Twins Toby and Corey and sister Izzy all have the rare and fatal condition juvenile Batten disease (CLN3). Children affected by juvenile Batten disease experience a wide range of symptoms including sight loss, tremors, epilepsy, problems with balance and walking, behavioural and psychological issues and, as the disease progresses, difficulties with chewing and swallowing. Tragically, the disease is fatal.
There are some treatments which can help with some of the symptoms of juvenile Batten disease, such as seizures. But there is no treatment that can slow the progression of the disease, which is caused by problems with a specific gene and is an inherited neurodegenerative condition.
Action Medical Research is funding research to investigate whether a medicine that’s already used widely to control blood pressure in adults might benefit children with juvenile Batten disease.