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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Hi Cheryl,
We all are on same boat. PRAYING TO GOD that HE bring the CURE for ALL OUR KIDS.

Raktim
The reason I open this forum is because we need to have many options on treatments because if an option is not good another will come.
Cheryl: BioMarin did state that they have other candidates that may be able to overcome the problems they had with BMN-195, so that indicates to me that they are still going ahead. However, you stated that this will off the menu for quite some time. Did you hear something that makes you think they won't be going ahead with this kind of research?

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. :(
No Terry I've heard nothing more than what everyone else heard, that this round didn't work. What I meant is that by the time they resculpt BMN-195 into something different, maybe better, it will most likely be too late for my child.

Terry Porcaro said:
Cheryl: BioMarin did state that they have other candidates that may be able to overcome the problems they had with BMN-195, so that indicates to me that they are still going ahead. However, you stated that this will off the menu for quite some time. Did you hear something that makes you think they won't be going ahead with this kind of research?

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. :(
ActionDuchenne's board has more info about this, they did talk to Biomarin's people yesterday. You can take a look. If not please let me know and I can copy and paste here. It's not very clear if BioMarin will continue utrophin upreg work at this time, but of course they state that the research should continue.
Please would you paste the information here thank you

Ofelia Marin said:
ActionDuchenne's board has more info about this, they did talk to Biomarin's people yesterday. You can take a look. If not please let me know and I can copy and paste here. It's not very clear if BioMarin will continue utrophin upreg work at this time, but of course they state that the research should continue.
Basically, one of AD trustees talked to BioMarin and the this is what he posted:

"I spoke to Bio Marin late last night and things are really still up in the air but the only thing they were pretty sure of is, IF they proceed with new targets it would take around 2 years to get to a phase 1 trial stage... not the best news... I do think that Utrophin is still a viable treatment but it will take longer than we thought and its route to market may be through Bio Marin or another company"



Alejandra Lagffer said:
Please would you paste the information here thank you

Ofelia Marin said:
ActionDuchenne's board has more info about this, they did talk to Biomarin's people yesterday. You can take a look. If not please let me know and I can copy and paste here. It's not very clear if BioMarin will continue utrophin upreg work at this time, but of course they state that the research should continue.
Other post was this:

"Like all parents and young men living with Duchenne this announcement leaves me deeply disappointed. In truth I'm gutted. But I would like to share with everyone some information from Biomarin and some thoughts on where we go from here.

Biomarin inform me that there was no difference between those taking a placebo and those taking the drug in terms of utrophin upregulation in their phase 1 trial. There are other factors too but simply the drug does not work and there is no point continuing the trial. We have to understand that 90% of drugs fail at clinical trial stages 1 and 2 and this failure certainly puts those figures into some perspective.

The development of this drug has been a hard road. We have been working with Kay Davies, Vastox, Summit and Biomarin funding and supporting this effort for several years. No drug company was interested in Kays work or Duchenne for that matter and so with Jon Tinsley and others she had to set up Summit to try to get the drug trialled. Biomarin finally agreed a partnership and has worked hard to get this well organised double blind trial underway.
Unfortunately we have not got the result we wanted.
Biomarin remain committed to supporting Duchenne drug development and we will have to wait and see if they develop more candidates. They have agreed to come to our conference in November in London to discuss the out comes of this trial and their future work.

We must understand the need for working with companies like Biomarin who can organise the sort of trials that can give us answers. Otherwise we continue to work on drugs or medicines in the lab and never get to conclusive human trials.

Action Duchenne has teamed up with Charley's fund and others in the USA to establish Pilot Trials Now - this will be a programme of pilot clinical trials to see if known drugs for other diseases could benefit children with Duchenne. The first trial is underway testing Viagra in the US. Action Duchenne has committed $250,000 already to further trials of other drugs. We want to do more.

Exon skipping drugs have passed through initial trials. Action Duchenne has funded and supported research in AO drug development. AVI, Prosensa, GSK are making rapid progress in this field. Action Duchenne and Duchenne Ireland are currently funding a multi skipping project at Oxford to see if we can use exon skipping for rarer deletions and duplications.

In the near future we hope to develop some of this research in China.

Other drugs and research is in the pipeline and some of these research groups and companies will present at our Conference in November.

When we established Action Duchenne in 2001 there were no human clinical trials taking place. That's zero trials anywhere in the world! Steroids were not being used by many clinicians.

We now have major pharmaceutical companies taking an interest and running trials. But it is not enough. Its not fast enough and we don't have enough money for all the research that needs to be done.

Cure. No compromise. I'm still here. Lets keep up the fight to cure Duchenne for every boy and girl. To do that we need to raise money and work together and work with other Duchenne charities and trusts as we are doing with Pilot Trials Now. "
So what's we've learnt this year is that MDX is not a reliable animal model... Good results in MDX do not mean much unfortunately. We'll see what happens with ACE-031...I did not see any data other than MDX and post-menoupausal women on that one. Sure hope that they have more data...

As for urtrophin upregulation, PTC has a compund that is "planned" to enter clinics in 2011. Not sure if PTC has resources to work on this before they figure out what to do with Ataluren though.
Professor Dame Kay Davies on Biomarin Trial

"The failure of BM195 Phase I trials is obviously very disappointing as it looked so promising in the mdx mice. However, we have new screens coming along which should provide new and better candidates for increasing levels of utrophin. We, like BioMarin, remain committed to utrophin upregulation for the therapy of DMD."

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