Why is a Batten disease natural history study important?

The purpose of a Natural History Study of Late Infantile Variant CLN5 and CLN7 Disease is to better understand two rare forms of Batten disease. It aims to:

  • Understand and document the medical history, clinical characteristics, progression of symptoms, and medical events in individuals with CLN5 and CLN7.
  • Provide greater understanding of clinically meaningful outcome measures to help inform endpoint selection for future clinical trials.