Mps 6 News Today

>> i thought about, like, whats 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 theyre going to come up with a better solution. i really do hope that i dont have to go through this again because this -- it was really stressful and overwhelm for me and my family and im sure for other families, so im hoping and praying that things will get better. >>...

Get this extended? >> i thought about, like, whats 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 theyre going to come up with a better solution. i really do hope that i dont have to go through this again because this -- it was really stressful and overwhelm for me and my family and im sure for other families, so im hoping and praying that things ...

Are real super heroic doctors who do that kind of work at the best childrens hospitals in the world, many of which are in the united states. but that work doesnt just happen by genius alone, right? it doesnt 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 u...

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 childrens 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...

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, thats the amazing thing...

Gene therapy for mps-6. we just need a few more years to bring this therapy to isabel so she doesnt need these infusions weekly that she can make her own enzyme, you know. it would be -- it will be an unbelievable tragedy if shes 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. shes been a contributor to your research, and im sure one of the real...

Gene therapy for mps-6, and we just need a few more years to bring this therapy to isabelle, so she doesnt need these infusions weekly, so she can make her own enzyme. and itll be an unbelievable tragedy if shes 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. shes been a contributor to your research, and im sure one of the real friends, important f...

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 f...

Gene therapy for mps-6, and we just need a few more years to bring this therapy to isabelle, so she doesnt need these infusions weekly, so she can make her own enzyme. and itll be an unbelievable tragedy if shes 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. shes been a contributor to your research, and im sure one of the real friends, important f...

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 m...