Gene Modified Stem Cells in MLD

We think that this girl can look forward to a normal life expectancy in a normal quality so clearly using gene modified stem cells in MLD is new. But we have a wealth of experience of using transplant from donors in similar diseases and what we see in those illnesses is that the effect on the condition is lifelong. We have every reason to be optimistic that the genemodified stem cells will also similarly dramatically change this young girl's life.