National Institutes of Health Seek Individuals to Participate in a Research Study
Researchers at the National Institutes of Health (NIH), in Bethesda, Maryland, seek individuals with CLN3...
Highly Promising 12-month results in the Phase I/II trial of Batten-1
Lyon, France – Austin, Texas, United States – 29 September 2023 – 7.30am – CET – Theranexus, a biopharmac...
Rare Sibling Stories Looking for Stories!
Rare Sibling Stories is looking for siblings to highlight on their platforms! They would love to feature...
NORD’s Batten Disease Patient Assistance Program
Having a rare disease is difficult. Adding in the complex care required to treat or manage that disease a...
BBDF Grants $60,000 Over 3-Years to Support Ineka Whiteman, Ph.D., Head of Research and Medical Affairs
BBDF grants $60,000 over 3-years to the BDSRA Foundation to support Ineka Whiteman, Ph.D. to serve as the...
Encouraging preliminary 6-month results in the Phase I/II trial of Batten-1 in Batten disease (CLN3)
Lyon, France – Austin, Texas, United States – 14 June 2023 – 7.30am CEST – Theranexus, a biopharmaceutica...
Expert Insights on Gene Therapy for International Batten Awareness Day
Neuronal ceroid lipofuscinosis (NCL) is a group of 13 rare inherited lysosomal disorders affecting the ne...
Theranexus and BBDF granted positive opinion by EMA for the design of Phase III trial to evaluate Batten-1 in CLN3 Batten disease
Lyon, France – Austin, Texas, United States – 7 June 2023 – 7.30 am CEST – Theranexus, a biopharmaceutica...
Families raised millions and handed rare disease therapies off to biotechs. But companies have backed out
When Julia Taravella’s two sons were diagnosed with an ultra-rare disease, she did something extraordinar...
Theranexus and BBDF win FDA approval on efficacy endpoints for the Phase III trial to evaluate Batten-1 in CLN3 Batten disease
Theranexus and BBDF win FDA approval on efficacy endpoints for the Phase III trial to evaluate Batten-1 i...