latest news on treatments to stop duchenne
even though exon skiping is a very promising treatment is not for all the kids with dmd if the error is in the first exons or in the last they are not ellegible if the exon dont match they are also not ellegible
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Permalink Reply by Alejandra Lagffer on
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for that reason we have to be open to other researchs for example the utrophin I think that is in the body of all of our kids it has to be activated I might be wrong. The thing is to find a solution for all our kids and not to loose hope
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Permalink Reply by Andrew Kerr on
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Your absolutely right. Exon skipping is not a cure anyways, and from what I can understand, we can't even be sure how much of an impact it will have. Sure, it changes DMD into BMD, but that still leaves the future very uncertain for our children. I think its important to keep going to pursue exon skipping, as it should buy at least some time. However, it obviously can't be the only direction research is going.
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Even if the exon skiping work out first it works just for a few kids because not all the boys are elegible for this treatment. There is the utrophin and the biostrophyn.
The utrophin is homologue with the distrophyn. I really dont know if this two would reverse the damage that leaves this illness. But maybe they would.
Alejandra Lagffer said:for that reason we have to be open to other researchs for example the utrophin I think that is in the body of all of our kids it has to be activated I might be wrong. The thing is to find a solution for all our kids and not to loose hope
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Permalink Reply by RAKTIM SINGH on
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Hopefully (prayers and fingers crossed), UTROPHIN and ACE-031 will work for all our kids. May be together they can work as a CURE also. We all are waiting for results of BMN-195. They said, we should hear someting in Q3 2010. There is conf call by Biomarin on 2nd AUG. Hopefully, they will say something postive about BMN-195 in that call.
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Permalink Reply by Bernardo A. Iriberri on
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Hi Raktim. Thank you very much for the news on BioMarin's conference call on August 2, although it looks like it's basically a discussion of their 2Q financial results.
RAKTIM SINGH said:Hopefully (prayers and fingers crossed), UTROPHIN and ACE-031 will work for all our kids. May be together they can work as a CURE also. We all are waiting for results of BMN-195. They said, we should hear someting in Q3 2010. There is conf call by Biomarin on 2nd AUG. Hopefully, they will say something postive about BMN-195 in that call.
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Yes we hope so to. I am sure that soon the cure will be found. I really hope the utrophin will work. And also read about the biostrophin even though they had a imune reaction they will work it out.
the utrophin has 75 exons the distrophyn 79 the utrophin is homologue with the distrophyn
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Would you posted the latest news on utrophin and what they say in the conference
thank you
RAKTIM SINGH said:Hopefully (prayers and fingers crossed), UTROPHIN and ACE-031 will work for all our kids. May be together they can work as a CURE also. We all are waiting for results of BMN-195. They said, we should hear someting in Q3 2010. There is conf call by Biomarin on 2nd AUG. Hopefully, they will say something postive about BMN-195 in that call.
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Permalink Reply by cheryl cliff on
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BioMarin just announced their utrophin didn't work in their Phase I clinical trial. Looks like this will fall off the menu for quite some time. :(
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Just read the news too about BMN 195. :(
http://phx.corporate-ir.net/phoenix.zhtml?c=106657&p=RssLanding...
I am not sure if I understand this. Did BMN 195 not produce the needed utrophin, or the utrophin was produced but it did not help, and the upregulation of utrophin is not a viable solution to over come the lack of dystrophin?
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I am terrible sorry to hear this news even though exon skiping is not for every kid my kid is one of those.
Thank you for the information.
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Permalink Reply by Jonathan on
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It is a setback, but they did say that they have a new candidate compound that overcomes the limitations of BMN 195. Its just a question of whether they do continue with this and how long it will take to get into trials.
cheryl cliff said:BioMarin just announced their utrophin didn't work in their Phase I clinical trial. Looks like this will fall off the menu for quite some time. :(
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And a question of who's kid can last while they fix this thing. Mine's already 12 in a few days and we watch duchenne take more and more of him. Hopefully another approach will finally work for all our guys and soon, Myostatin Inhibitor, Exon Skipping are next on the menu.
Jonathan said:It is a setback, but they did say that they have a new candidate compound that overcomes the limitations of BMN 195. Its just a question of whether they do continue with this and how long it will take to get into trials.
cheryl cliff said:BioMarin just announced their utrophin didn't work in their Phase I clinical trial. Looks like this will fall off the menu for quite some time. :(
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