Page 2 - Mps 6 News Today : Breaking News, Live Updates & Top Stories | Vimarsana

Gene therapy for mps-6. we just need a few more years to bring this therapy to isabel so she doesn't need these infusions weekly that she can make her own enzyme, you know. it would be -- it will be an unbelievable tragedy if she's taken off enzyme and is not able to reach this permanent solution. >> i have to believe that assess bell is more than just a patient to you after almost 20 years now of treatment and working together. she's been a contributor to your research, and i'm sure one of the real friends, important friends in your life. >> she is. it's a small community. we only have 50 to ...

Gene therapy for mps-6, and we just need a few more years to bring this therapy to isabelle, so she doesn't need these infusions weekly, so she can make her own enzyme. and it'll be an unbelievable tragedy if she's taken off enzyme and is not able to reach this permanent solution. >> i have to believe that isabelle is more than just a patient to you after almost 20 years now of treatment and working together. she's been a contributor to your research, and i'm sure one of the real friends, important friends in your life. >> she is. it's a small community. we only have 50 to 100 patients in the ...

A unique therapy. we can mention -- was one of the inventors of this therapy when it was first brought to the human for mps-1 and he helped move this forward for mps-6, which was the second disease that had therapy developed in this group. so it was a tremendous break through to be able to give these patients back the missing enzyme. and it was a breakthrough of new science, genetics, all of the ability to do gene therapy within cells and make this protein that we can infuse each week. and the future is open. i mean, that's the amazing thing that she's healthy and bright and vigorous and we're...

Gene therapy for mps-6, and we just need a few more years to bring this therapy to isabelle, so she doesn't need these infusions weekly, so she can make her own enzyme. and it'll be an unbelievable tragedy if she's taken off enzyme and is not able to reach this permanent solution. >> i have to believe that isabelle is more than just a patient to you after almost 20 years now of treatment and working together. she's been a contributor to your research, and i'm sure one of the real friends, important friends in your life. >> she is. it's a small community. we only have 50 to 100 patients in the ...

We can mention -- was one of the inventors of this therapy when it was first brought to the human for mps-1 and he helped move this forward for mps-6, which was the second disease that had therapy developed in this group. so it was a tremendous break through to be able to give these patients back the missing enzyme. and it was a breakthrough of new science, genetics, all of the ability to do gene therapy within cells and make this protein that we can infuse each week. and the future is open. i mean, that's the amazing thing that she's healthy and bright and vigorous and we're within a few year...