Posted on: 24 October 2019, source: wbur
It's a very early report, from just two patients, only a few months after treatment. But UMass Medical School Dean Terence Flotte this week shared at a conference what could be landmark news about a terrible genetic disease: Two young patients with Tay-Sachs disease showed no ill effects from a new gene therapy that aims to correct the defect at the heart of the disease. One of them, treated at just 7 months, has appeared to stabilize instead of following the typical quick slide toward death by age 4. "It seems right now that she's not degenerating," Flotte said. "But I would say it's too early to say that definitively."