The data is interesting, the clips of the kids is astonishing. Also that a 12 and now 14 yr old kid remains stable given the stage of the disease. And that seems to be the main point: I see no real progress (but that is subjective) but I do see regardless of age that there is no detoriation. So a new therapy that is not depending on the same or similar vector for delivery might help these kids even more.
The data is interesting, the clips of the kids is astonishing. Also that a 12 and now 14 yr old kid remains stable given the stage of the disease. And that seems to be the main point: I see no real progress (but that is subjective) but I do see regardless of age that there is no detoriation. So a new therapy that is not depending on the same or similar vector for delivery might help these kids even more.