Research abstracts, publications, NTSAD initiatives, and Tay Sachs Gene Therapy Consortium (TSGT)
The goal of the subcommittee is to evaluate clinical trials or other proposed therapies for Tay-Sachs, GM1, Sandhoff, Canavan and related diseases to educate families regarding trials and how to assess a proposed new or untested therapy. Papers written on different topics of therapies and/or clinical trials will be saved in this folder.
Semi-annual progress reports published by TSGC on gene therapy research.