Classifieds

Much of the NINDS research on Batten disease focuses on developing a better understanding of the disease, gene therapy, and the development of drugs to treat the disorder. Scientists are using a modified safe virus to deliver a functioning gene to the brain with hopes the replacement gene will take over or restore the mutated gene’s normal function. Using an animal model, NINDS-funded scientists are combining gene therapy with bone marrow transplant as a therapy for infantile Batten disease.  NIH researchers have identified a potential new drug to treat infantile Batten disease that uses a non-toxic molecule to prevent the harmful cellular residue buildup. Among other research, scientists are looking at the development of new molecules to treat Batten and a variety of neurological disorders. Other scientists hope to identify biomarkers–biological signs that may indicate the presence or progression of a disease–to better understand the disease and possibily develop new treatments. The NINDS helps fund the Lysosomal Diseases Network, a combined network of research centers, clinical investigators, patient advocacy groups, and other interested parties that advocate for research on diagnosing, managing, and treating lysosomal and related diseases, including Batten disease....