RT001 to treat neurodegeneration—Case Study: Improvement in a single patient with Late Onset Tay-Sachs Disease (LOTS)

Objective: We report on the use of RT001 in a single patient with LOTS.

Conclusions:

1. D-LA is elongated to D-AA; both achieve significant plasma levels and RBC membrane incorporation within 1 month of administration.

2. Early efficacy signs of inhibition of disease progression and some regression were seen.

3. RT001 was well tolerated with no toxicities reported.