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  • New Mutation Found in Newborn with Congenital Neuronal Ceroid Lipofuscinosis, Case Study Reports
  • ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
  • #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
  • Lack of Progranulin Protein Sheds Light on Molecular Features of Batten Disease, Study Shows
  • Study Examines Promising Advances in Gene Therapies to Treat Batten Disease
  • More Reliable Tools Needed to Research CLN3 Protein in Batten Disease, Study Shows
  • Brineura Helps Children with CLN2 Retain Motor, Language Skills, Trial Data Show
  • #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12
  • ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease
  • Several Compounds Found to Correct Brain Defects in Mouse Model of CLN3 Disease
  • Hair-like Structures on Cells Play Role in Batten Disease Development, Mouse Model Shows