Beyond Batten Disease Foundation is excited to share the latest accomplishment on our journey to find a treatment for juvenile Batten disease. Six young adults over 17 years old have been successfully treated with miglustat over the past year. The results show that the drug is safe to use at the maximum dose. These results, in conjunction with a recent meeting with the FDA, allow us to proceed to the Phase III efficacy trial in 2023. Your support has been critical in the development of this program. We are one step closer to having the first ever treatment for CLN3 disease. Thank you for making this possible.
Join us in our mission to find a cure for Batten disease and prevent hundreds of similar rare genetic diseases that claim the lives of thousands of children every year.
New Advance to the Theranexus and BBDF Batten-1 Program for Juvenile Batten Disease (CLN3)
Beyond Batten Disease Foundation ⁄ BBDF Blog ⁄ Research ⁄ New Advance to the Theranexus and BBDF Batten-1 Program for Juvenile Batten Disease (CLN3)