News

A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured in several media platforms, including American Way…

New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that combined treatment of the spinal cord and the brain has significant therapeutic effects. The study titled, “Synergistic effects of treating the spinal cord and brain in CLN1 disease,” was published…

Gov. Tom Wolf Pennsylvania  Gov. Tom Wolf recently signed House Bill 239, establishing the Rare Disease Advisory Council. The bill, was introduced May 24 by state Rep. Marcy Toepel, R-Montgomery County, who said the bill is an outgrowth of contact with people in her district who…

European and U.S. regulators have released a white paper on ways to develop innovative therapies for Gaucher disease that could also apply to Batten disease and other rare children’s disorders. The goal of the European Medicines Agency and U.S. Food and Drug Administration paper is to spur novel approaches to creating treatments.

Abeona Therapeutics’ ABO-201 program was designed at orphan drug by the U.S. Food and Drug Administration (FDA) as a potential gene therapy treatment for juvenile Batten disease. A clinical study of the therapy in patients is in the planning stages. ABO-201 therapy uses a virus to deliver a normal copy of…

Kennedy Hansen Kennedy Hansen had already lost her vision and most of her motor skills to Batten disease by 2013, but when the head of her cheerleader squad went around a circle at practice one day asking each member to name their life…

Stem cell models of lysosomal storage disorders (LSDs), including Batten disease (BD), could significantly add to knowledge of disease biology and help develop improved therapies, a new review highlights. The study, titled “Induced pluripotent stem cell models…

The fat-lowering therapy gemfribrozil decreased disability and increased the lifespan of mice with a form of Batten disease that strikes children, according to a preclinical-trial study. The finding suggests the high-cholesterol drug could be used to treat children with Batten, which scientists call Neuronal Ceroid Lipofuscinosis, or NCL. The research also indicated…

Although Brineura (cerliponase alfa) is now approved both in Europe and the U.S. to treat CLN2 type Batten disease, its clinical trial path is far from over. As the approval rested on a small Phase 1/2 trial, an ongoing extension study (NCT02485899) is gathering additional safety and efficacy data. Meanwhile, the…

 Brineura (cerliponase alfa), by BioMarin, is now approved in Europe to treat neuronal ceroid lipofuscinosis type 2 (CLN2), one of a group of disorders known as Batten disease. The therapy covers all ages from birth. The marketing authorization granted by the European Commission (EC) follows a positive review by…