Families and Researchers Collaborate in Advancing Therapy: The Role of NTSAD, Together with Parents, in Driving Toward Treatments.

‘‘IS THERE A TREATMENT?’’ are always the first words uttered when a parent or relative of a newly diagnosed child contacts National Tay-Sachs & Allied Diseases Association (NTSAD) upon hearing the devastating news that their loved one was diagnosed with one of the life-limiting diseases supported by NTSAD. The simple answer is always the same: ‘‘There are no treatments.’’ But first we try to inject hope by telling them about the ongoing research, especially in gene therapy, as it has received much NTSAD research grant support for a number of years and has produced promising results in animal models. Investigational new drug (IND)-enabling studies are in process; we remain optimistic for reaching the next major milestone and then hopefully demonstrating efficacy in affected children or adults.