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
Replies to This Discussion
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Permalink Reply by Bernardo A. Iriberri on
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Well, this really hits me hard, as I became a sort of fan of Kay Davies almost ten years ago, without ever meeting her, when I started reading about her work in the area of utrophin upregulation. I had placed high hopes on that line of research, but then I became almost obsessed with PTC124 (my son has a premature stop codon type of mutation), until early this year the whole thing fell down. When the Ataluren trials were stopped I started placing my hopes afain on BioMarin and the utrophin reasearch, and now we have this. What can one say? Thank you all for the information
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This ends BioMarin's involvment.
A quote from Summtplc.com
"BMN-195 subsequently entered into Phase I clinical trials in January 2010 before BioMarin took the decision to discontinue development of this compound in August 2010 citing pharmaceutical and pharmacokinetic challenges related to plasma concentrations of the compound. BMN-195, and all associated intellectual property, will now transfer back to Summit.
Summit remains committed to working in DMD believing that use of an appropriate formulation has the potential to produce a viable medicine and is investigating commercial opportunities to support the future development of BMN-195."
http://www.summitplc.com/default.aspx
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Permalink Reply by Alejandra Lagffer on
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Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes-called meganucleases-with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells.
Read more: Repair Possible of Gene Responsible for Duchenne Muscular Dystrophy http://www.medindia.net/news/Repair-Possible-of-Gene-Responsible-fo...
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Permalink Reply by Cheri Gunvalson on
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Alejandra Lagffer said:Professor Tremblay's team partnered with Cellectis, a French firm specializing in genome engineering, in order to design enzymes-called meganucleases-with the ability to correct the dystrophin gene. During in vitro testing, the researchers inserted genes coding for a variety of meganucleases into human muscle cells.
Read more: Repair Possible of Gene Responsible for Duchenne Muscular Dystrophy http://www.medindia.net/news/Repair-Possible-of-Gene-Responsible-fo...
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This is exciting. My big question is this a systemic method of delivery or does it involve many many many injections such as in Dr Tremblays previous research? I was fortunate to meet Dr Tremblay when he chaired the first NIH peer review that I was on for the first round of selection of the Wellstone centers. He is very passionate for his research. Like all of you I hope and pray it works but I could not subject our son to injecting each muscle.
http://www.medindia.net/news/Repair-Possible-of-Gene-Responsible-fo...
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Permalink Reply by Jeff Sobel on
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I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said: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.
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In the studies for how long do they do the tests on MDX before taking to clinical trials. Do they tried the Ace 31 with kids with duchenne.
Jeff Sobel said:I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said: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.
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I also was waiting for the results of the utrophin I thought that this was going to be ok since the utrophin is already in the body because the other studies had not succeded because of the inmune reaction
but we have to keep waiting for the best to come
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Permalink Reply by Ofelia Marin on
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I agree. I think that they also noted that long term steroid trt increases fibrosis in the heart in mdx.
Problem is that experiments in other animal models (dogs etc.) are much more expensive. I wonder if it would be a better idea to also use those dKo mice (no dystrophin or utrophin). Either way promissing new drugs will be tested in humans and hopefully some will prove effective in the near future.
Jeff Sobel said:I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said: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.
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There is a clinical trial in Canada testing ACE-031 in DMD boys 4 years or older.
http://www.clinicaltrials.gov/ct2/show/NCT01099761?term=DMD+ACE&...
Alejandra Lagffer said:In the studies for how long do they do the tests on MDX before taking to clinical trials. Do they tried the Ace 31 with kids with duchenne.
Jeff Sobel said:I would add that a corollary of your statement, which I agree with, is that negative effects of compounds on mdx mouse also do not tell us what the effect might be in duchenne. In particular, I note recent mouse lab research indicating that l-arginine might increase fibrosis of skeletal and cardiac muscle. This research tells us nothing regarding whether l-arginine, which I continue to have my son take daily, is beneficial or harmful in the long run. These mouse articles simply discuss science experiments, and we have yet to see successful translation to humans. It seems that the more we learn, the more difficult the Duchenne problem becomes. Nonetheless, we must push forward.
Ofelia Marin said: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.
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Permalink Reply by R. Gardiner on
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We were just told that the GSK exon skipping trial has been delayed in Canada and the US as well, for approximately 8 weeks. We were told it was because of logistical reasons, whatever that is suppose to mean. Our son is eligible to take part in the trial in London, Ontario but now we are worried that it may not take place here. We heard that Dr. Mendell stated
Columbus Ohio was going to be the only North American trial sight. Has any one else heard this? Does anyone have more information about the reason for delaying these trails?
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