Over the last 9 years, Beyond Batten Disease Foundation (BBDF) has funded research for a treatment or cure for juvenile Batten (CLN3) disease. That research has led to a medical discovery: a treatment that slows the progression of the disease in Batten models.
We are now laser-focused on gaining approval from the US FDA to conduct a clinical trial to treat children with CLN3 disease. If successful, the regulatory pathway we have chosen will lead to approval of the new medicine for all patients, wide distribution, make the drug eligible for insurance coverage, and open the door for treatment of other forms of Batten and lysosomal storage diseases.
We need your help! Anecdotal information from the patient community about their experiences can be very helpful in supporting our case if included as part of our submission package to the FDA. Please take 2 minutes to fill out this short survey
to provide important information about your personal experiences, if any, with Trehalose and/or Miglustat.
The survey is confidential and will be anonymous unless you choose to share your contact information. Participating in the survey will in no way affect eligibility for this or any other trial.
Please let us know if you have questions or would like additional information.
With gratitude for all you do for your own family and the broader Batten community. We are grateful for your partnership in this effort.
Look forward to hearing from you!