Posts by panini
1 - 3 of 7 Posts
Joined
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5,290 Posts
Well we are on our way to saving quite a few kids. The two Holland girls have finished their clinical trial and are getting better everyday. We have most of the results from the trials and we should announce them at the international MPS meeting in Paris the begining of next year.
I didn't want to post ahead of time but this is the foundation I'm always talking about. We have been on the board since the beginning. We are a group of 6 families. We just finished our annual golf gala in Austin where we raised 500,000. dollars. We use every penney that is made for research.
The enzyme is being given to the kids weekly for six hrs. Unfortunately we can't seem to get the enzyme past the BBB blood brain barrier into the brain.
We now have to raise 6-10 million to develope a gene replacement therapy. We have the gene, but just need to figure out what type of virus will transport it to the brain.
Sorry, long, this is our second life. I know this is not the place for this but if anyone else is interested ryanfoundation.org
jeff
I didn't want to post ahead of time but this is the foundation I'm always talking about. We have been on the board since the beginning. We are a group of 6 families. We just finished our annual golf gala in Austin where we raised 500,000. dollars. We use every penney that is made for research.
The enzyme is being given to the kids weekly for six hrs. Unfortunately we can't seem to get the enzyme past the BBB blood brain barrier into the brain.
We now have to raise 6-10 million to develope a gene replacement therapy. We have the gene, but just need to figure out what type of virus will transport it to the brain.
Sorry, long, this is our second life. I know this is not the place for this but if anyone else is interested ryanfoundation.org
jeff
Joined
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5,290 Posts
Wendy,
No not all of the children are getting the enzyme. The FDA has decided to have the second trial which has ended and the results are even better then the first round. We are hoping for the ERT to be approved early next year. At which time we can maybe get a little more exposure. We're fielding e-mails from all over the world from persons afflicted with the disease and hearing about us for the first time. Some of the kids are not even diagnosed properly. We have heard from persons with children that have been attending Childrens Hospitals where the families and the doctors are just waiting for the end. Don't know of us. Immagine the rural areas! Like I said, on the outside it seems like we are a big foundation but the reality is that Mark has given up his career with the police department , no possibility of advancing with all his time off. There are six other families that spend, well anyway.
Marm,
I don't have to explain the politics to you about research. Two years ago after isolating the enzyme we needed the help to bring it to the FDA so a small bio-tech company decided to fund us with 5 million dollars to get the enzyme approved. You know that right around the corner in medicine is 6-7 yrs. It gets frustrating sometimes. We have now granted some of our monies to other researchers to develope the gene. For GRT. This will be the most logical way to get the enzyme to the brain.
Your're right about the global community, If we accomplish what we have planned this will not only help our disease but many others. I am surrounded by heros in my life. It is a power not with us that brought this doctor into our lives that built his lab basically in the parking lot of UCLA with the first 40,000. we raised. I'll stop. as you can see where our passion lies.
Thanks for all of your support. You know food has become a big part of our event, we auctioned 8 dinners for 8 persons @2000. ea. Maybe next year we can do something involving other cities.
Believe it or not not one penny is spent on anything but research, we solicate donations for everything including copy paper,web-site,paperclips,printing etc. Even those of us on the board pay for our gala just as the rest. gtg
jeff
No not all of the children are getting the enzyme. The FDA has decided to have the second trial which has ended and the results are even better then the first round. We are hoping for the ERT to be approved early next year. At which time we can maybe get a little more exposure. We're fielding e-mails from all over the world from persons afflicted with the disease and hearing about us for the first time. Some of the kids are not even diagnosed properly. We have heard from persons with children that have been attending Childrens Hospitals where the families and the doctors are just waiting for the end. Don't know of us. Immagine the rural areas! Like I said, on the outside it seems like we are a big foundation but the reality is that Mark has given up his career with the police department , no possibility of advancing with all his time off. There are six other families that spend, well anyway.
Marm,
I don't have to explain the politics to you about research. Two years ago after isolating the enzyme we needed the help to bring it to the FDA so a small bio-tech company decided to fund us with 5 million dollars to get the enzyme approved. You know that right around the corner in medicine is 6-7 yrs. It gets frustrating sometimes. We have now granted some of our monies to other researchers to develope the gene. For GRT. This will be the most logical way to get the enzyme to the brain.
Your're right about the global community, If we accomplish what we have planned this will not only help our disease but many others. I am surrounded by heros in my life. It is a power not with us that brought this doctor into our lives that built his lab basically in the parking lot of UCLA with the first 40,000. we raised. I'll stop. as you can see where our passion lies.
Thanks for all of your support. You know food has become a big part of our event, we auctioned 8 dinners for 8 persons @2000. ea. Maybe next year we can do something involving other cities.
Believe it or not not one penny is spent on anything but research, we solicate donations for everything including copy paper,web-site,paperclips,printing etc. Even those of us on the board pay for our gala just as the rest. gtg
jeff
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