Parents of two sons, one of whom has CLN2 (Batten disease) and is receiving enzyme replacement therapy, share how they think about the future. “We are optimistic. There are other treatments coming down the pike, such as gene therapy.  And in the meantime, we’re helping the children that come later. … Hopefully we’re the last family that will have been told their child is going to die.” Mom describes the shift in the disease community now that there is an approved treatment and gene therapy trials on the horizon. The outlook is so different now.”