RE:RE:What Hunter Syndrome Could Be Worth: "reached therapeutic levels in the CNS and significantly decreased the number of lysosomal storage vacuoles in the cytoplasm of brain cells."
Read more at https://www.stockhouse.com/news/press-releases/2015/07/31/bioasis-announces-the-successful-delivery-to-the-cns-of-an-enzyme-used-to-treat#yIsB50KFl0BS1OgU.99
While the periphery was normalized with \bti's technology, it may be useful to know what percentage of lysosomal storage vacuoles in brain cells remained in the treatment group vs. placebo so we know how much work is left to do. Also, has BTI established a dose-response relationship for its drug that shows dosing alone can lead to meaningful increases in delivery over the regimen chosen in the study. The absence of data in the news release may not impress the market.