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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…

Treatment with anti-inflammatory drugs fingolimod (Gilenya) and teriflunomide (Aubagio) may potentially fight brain inflammation and neuronal death in patients with neuronal ceroid lipofuscinoses (NCLs or CLNs), according to new reserch. CLNs are a group of disorders of which Batten disease is the most common. Aubagio, marketed by…

Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, was found to improve life expectancy and motor function in mice with late infantile neuronal ceroid lipofuscinosis (late infantile NCL, or LINCL), which belongs to a group of disorders collectively referred to as Batten disease. The study, “Gemfibrozil, Food and Drug Administration-approved lipid-lowering drug,…