About Us
BDSRA is dedicated to funding research for treatments and cures, providing family support services, advancing education, raising awareness, and advocating for legislative action. Founded in 1987, by parents seeking to build a network for those diagnosed with Batten disease, BDSRA is now the largest support and research organization dedicated to Batten disease in North America. BDSRA believes that to effectively unravel the mysteries of Batten disease, the worlds of medical science, research, and families must work together toward a common goal: discover treatments and cures while assuring a better quality of life for those living with the disease.
Recent News
CLN2 Batten Disease Update from Tern Therapeutics
More positive news for our CLN2 community this week, with Tern Therapeutics announcing that its investigational gene therapy, TTX-381, for the treatment of ocular manifestations of CLN2 disease, has been selected by the FDA to participate in the Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) Program. TTX-381 already holds Regenerative Medicine Advance Therapeutics (RMAT) and Fast Track designations, and this new selection will help Tern work closely with the FDA to speed manufacturing readiness and support earlier [...]
CLN2 Batten Disease Update from Latus Bio
Latus Bio announced today clearance by the U.S. Food and Drug Administration (FDA) of the Company’s Investigational New Drug (IND) application for LTS-101, a gene therapy candidate that’s intended to treat the CNS manifestations of CLN2 disease. The FDA has also granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations for LTS-101. Full Press Release Giving Tuesday Update We made early progress! As of this morning, we were nearing $300 on the way to our [...]
