Non-Interventional Feasibility Study of a Real-World Data Trial of Patients Using Miglustat Off-Label for CLN3
The Beyond Batten Disease Foundation, a non-profit organization founded in 2008 to eradicate juvenile Batten disease, in partnership with Engage Health, is conducting a research study to better understand the off-label use of Zavesca® (miglustat) in individuals with CLN3 Batten Disease. The Non-Interventional Feasibility Study of a Real-World Data Trial of Patients Using Miglustat Off-Label for CLN3 study is being conducted to gather insights from existing assessments and results (including (visual acuity data) to help inform upcoming clinical trials. Participation involves a short online survey and uploading relevant documents, which takes about 30 minutes to complete. Any caregiver of, or patient diagnosed with CLN3, who is able to read, speak, and understand English and is willing to upload or their visual acuity assessments is welcome to participate. To begin, follow the link here to participate.