Batten Disease is a lysosomal storage disorder, interfering with cells’ ability to break down wastes. The build-up of waste or, lipofuscin, causes cell death. There are multiple types of Batten.
With CLN2, Late Infantile, children are healthy and develop normally for the first few years of life. CLN2 is currently the only form of Batten disease that has an FDA approved treatment — enzyme replacement therapy that was approved by the FDA in April 2017. It was approved to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric CLN2 Batten disease patients 3 years of age and older. It is available commercially through several hospitals across the U.S.
(excerpted from BDSRA.org)
Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer perspective on coping with rare and/or life-limiting conditions. They also each represent a particular moment in time.
If you are considering a clinical trial for your child, please also see the Courageous Parents Network Guided Pathway on Clinical Trials.