Joana Fernandes, PhD,  —

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…

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…