An even rarer type of Tay-Sachs is called “late onset” or “adult onset.” It can be hard to diagnose. Like the version of the disease that affects infants, Tay-Sachs that starts later in ...

Patient Cenzy Thornton (who is not the baby treated with gene therapy) and her father at the 2017 Annual Family Conference of the National Tay-Sachs and Allied Disease Association. (Courtesy NTSAD ...

The youngest Tay-Sachs patients – that is, the ones with the fastest buildup of GM2 – typically have less than 0.1 per cent of normal HexA activity in their brains. Yet adult patients still ...

the research team has identified an existing FDA-approved drug that could significantly improve quality of life for affected patients and their families. "Sandhoff and Tay-Sachs are devastating ...

After more than 14 years of development, two babies have received the first-ever gene therapy for Tay-Sachs disease ... which usually isn’t possible for patients at this age.

Results: Thirty patients (67% male, age range 18–56 years) with late-onset Tay-Sachs disease were enrolled ... and tolerability of miglustat in adult patients with G M2 gangliosidosis.

Here, doctors produce a small molecule in Tay-Sachs patients that can help correct the faulty hexosaminidase A enzyme. Meanwhile, the jury is still out on trials currently being conducted for the ...

Taysha Gene Therapies Inc (NASDAQ: TSHA) reported initial serum β-hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, two forms of GM2 ...

These companies are at the forefront of developing cutting-edge therapies to improve patient outcomes. The drug pipeline for Tay-Sachs includes promising candidates such as venglustat (GZ402671 ...

the research team has identified an existing FDA-approved drug that could significantly improve quality of life for affected patients and their families. "Sandhoff and Tay-Sachs are devastating ...

Tay-Sachs disease, the degenerative and always fatal neurological genetic disease that is most commonly carried by Ashkenazi Jews, has been recognized even by ultra-Orthodox rabbis as a legitimate ...