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

>> i thought about, like, what's going to happen in two years, but, you know, i feel like sharing my story, you know, in d.c. in september, the situation about the status, medical deferred action. my hope is in two years hopefully they're going to come up with a better solution. i really do hope that i don't have to go through this again because this -- it was really stressful and overwhelm for me and my family and i'm sure for other families, so i'm hoping and praying that things will get better. >> well, isabel, i want to thank you also on behalf of all of the families of -- and the patients...

Get this extended? >> i thought about, like, what's going to happen in two years, but, you know, i feel like sharing my story, you know, in d.c. in september, the situation about the status, medical deferred action. my hope is in two years hopefully they're going to come up with a better solution. i really do hope that i don't have to go through this again because this -- it was really stressful and overwhelm for me and my family and i'm sure for other families, so i'm hoping and praying that things will get better. >> well, isabel, i want to thank you also on behalf of all of the families of ...

Are real super heroic doctors who do that kind of work at the best children's hospitals in the world, many of which are in the united states. but that work doesn't just happen by genius alone, right? it doesn't happen with, you know, capes and superpowers. in order to develop cures for rare diseases, what you need is the ability to work with patients who have those rare diseases. and by definition of the word rare, there are not all that many of those patients, and so doctors, for example, at the uc san francisco children's hospital in northern california, doctors who are looking to test a pot...

Know, capes and superpowers. in order to develop cures for rare diseases, what you need is the ability to work with patients who have those rare diseases. and by definition of the word rare, there are not all that many of those patients, so doctors, for example, at the uc san francisco children's hospital in northern california, doctors who are looking to test a potential treatment for a devastating rare disease called mps-6 where kids are born without an enzyme they need to live, doctors wanting to develop a treatment for that rare disease, well, they found a little 7-year-old girl named isab...

One of the inventers of this therapy when it was first brought to the human for mps-1. he helped move this forward for mps-6, which was the second disease that was -- had therapy developed in this group. it was a tremendous breakthrough to be able to give the patients back the missing enzyme. 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 is that isabel is healthy and bright and vigorous and we're within a few years of being able to do...