A small molecule inhibitor of the sortilin protein reduced cellular waste product accumulation and eased neuroinflammation in mouse…
Articles by Lindsey Shapiro, PhD
Digital biomarkers are a feasible way to remotely monitor motor function in people with rare neurological diseases, but more work…
CLN5-targeted gene therapy safely preserved clinical function, prolonged survival, and prevented brain tissue atrophy in a sheep model of CLN5…
Researchers have developed a new minipig model of juvenile Batten disease that recapitulates some key features of the human…
Researchers have identified new cellular roles for the CLN3 protein, the loss of which is the cause of juvenile…
Researchers have discovered a new naturally occurring nonhuman primate model of late-infantile Batten disease, or CLN2 disease. The primates,…
The U.S. Food and Drug Administration (FDA) has cleared the design of a pivotal Phase 3 trial to test…
The Batten Disease Support and Research Association (BDSRA) Australia has announced the three winners of its 2022 Batten…
Structural and functional changes in the retinal pigment epithelium (RPE) — a cell layer in the eye that nourishes the…
Some cases of Batten disease may present with clinical features of Rett syndrome, including Rett’s hallmark repetitive hand…