• Abeona Will Give Update on Its Gene Therapy Programs in R&D Day Set for October
  • Batten Disease Clinical Trials Enrolling Patients to Evaluate Gene Therapies
  • Polaryx’s Treatment Candidate for LINCL Granted FDA Orphan Drug Status
  • Charlotte and Gwenyth Gray Foundation Funds Clinical Trial for Rare Batten Disease Variant
  • National Organization for Rare Disorders Gives Batten Disease Community a Voice in Washington
  • Current Batten Therapeutics Targeting Brain Not Enough to Stop Disease in Other Organs, Canine Study Suggests
  • CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study
  • North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs
  • Infantile Batten Disease May Begin in Spinal Cord Rather Than Brain, Study Suggests
  • Young Batten’s Patient Inspires Lawmakers to Create Rare Disease Advisory Council
  • EU and US Regulators Offer Recommendations on Developing Therapies for Children’s Diseases
  • FDA Marks Potential Gene Therapy for Juvenile Batten Disease as Orphan Drug to Speed Development