Dr. Hoffman making headway on the research with the puppies!

PLEASE CHECK OUT THIS ARTICLE!!! http://insciences.org/article.php?article_id=3331

WASHINGTON, DC—Genetic researchers at Children’s National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of “multiple exon-skipping” to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as a “DNA band-aids” to skip over the parts of the mutated gene that block the effective creation of proteins. Be sure to check out the videos because they will show you what a puppy looks like without treatment and then a puppy one with treatment. The biggest part you'll notice, is how much better the treated puppy is physically capable of running!

Non-treated 7 month old pup: http://childrensnational.org/files/video/research/MuscularDystrophy...

Treated 7 month old pup: http://childrensnational.org/files/video/research/MuscularDystrophy...

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WOW! Could you imagine if it was that easy a treatment for our kids. It just about made me cry when I saw the difference. You couldn't tell that puppy had any kind of disease, never mind DMD. The next step is our boys...
Absolutely!!! This is the first time I've seen the videos published. I had seen them a year ago when I went to a presentation by him. It was very exciting then and I'm very excited now to see how far the news of this great research has come!!!!

One thing to note that the article did not mention. The is not really a cure in the sense of reversing any damage that has been done so far. This treatment is extremely effective but must be done as early as possible in the disease progression progress.

Joshua's mom said:
WOW! Could you imagine if it was that easy a treatment for our kids. It just about made me cry when I saw the difference. You couldn't tell that puppy had any kind of disease, never mind DMD. The next step is our boys...
Yes, but if it means that a teenager can still use a keyboard, brush his teeth, and feed himself, that's no small thing. Do you know whether this is a PMO or a PPMO agent?

Liisa Underwood said:
Absolutely!!! This is the first time I've seen the videos published. I had seen them a year ago when I went to a presentation by him. It was very exciting then and I'm very excited now to see how far the news of this great research has come!!!!

One thing to note that the article did not mention. The is not really a cure in the sense of reversing any damage that has been done so far. This treatment is extremely effective but must be done as early as possible in the disease progression progress.

Joshua's mom said:
WOW! Could you imagine if it was that easy a treatment for our kids. It just about made me cry when I saw the difference. You couldn't tell that puppy had any kind of disease, never mind DMD. The next step is our boys...
Yokota et al. used unmodified Morpholino oligos from Gene Tools, LLC.

Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S, Hoffman E. Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs. Ann Neurol. 2009 Mar 13. [Epub ahead of print]

http://www.ncbi.nlm.nih.gov/pubmed/19288467
These are PMOs, cannot be delivered to the heart. They have this quote: "However, the treatment was unable to prevent deterioration of the animals’ hearts. The researchers theorized that the muscles of the heart are less porous than the skeletal muscles, and did not absorb sufficient quantities of the morpholinos to curb the deterioration. "
Jeff Chamberlain from UW proved that when only the skeletal muscles are treated the heart problems start much earlier.

Also, please note the very high dose used 120-200 mg/kg weekly or biweekly (30-50 times higher than the highest dose in the UK trails skipping exon 51).




Paul Cliff said:
Yes, but if it means that a teenager can still use a keyboard, brush his teeth, and feed himself, that's no small thing. Do you know whether this is a PMO or a PPMO agent?

Liisa Underwood said:
Absolutely!!! This is the first time I've seen the videos published. I had seen them a year ago when I went to a presentation by him. It was very exciting then and I'm very excited now to see how far the news of this great research has come!!!!

One thing to note that the article did not mention. The is not really a cure in the sense of reversing any damage that has been done so far. This treatment is extremely effective but must be done as early as possible in the disease progression progress.

Joshua's mom said:
WOW! Could you imagine if it was that easy a treatment for our kids. It just about made me cry when I saw the difference. You couldn't tell that puppy had any kind of disease, never mind DMD. The next step is our boys...
Question here....I recall that an Italian Group had successfully done exon skipping with golden retrievers and was successful. Does anyone recall that? It was probably a couple of years ago. What happened there?

Also, I don't want to sound negative, but getting this into humans and approved by FDA will take considerable amount of time. More time than my 6 year has I am afraid....And, it was my understanding that the FDA was going to regard each cocktail as a unique RX and require testing....

Char Burke
Just like you Char, my son is 6 years old and he is slowly running out of time.
My only hope is Biomarin, but when is the question????
Char,

Yes, each exon will be tested. But my understanding is that as these first groups show positive results, then the process will speed up. At the current rate, it would take approx 400 +/- years to test each one. Obviously, there's no time for that. So, after the first few show positive results, then there would be multiple studies going on simultaneously with different exons. And, because of previous positive results, drug to market should be expedited because of those successes.

But, for exon skipping, i don't count on it to work for every child. There are plenty of cases of BMD that progress as rapid as DMD.

That's why my interest is in a cocktail of drugs.

As for Italy, I believe you are talking about Dr. Cossu. His study was some kind of meso(or immuno)-angioblast or something. I have been out of touch with research for quite sometime now, so my recollection could be incorrect. Anyway, if i remember correctly, it was basically a super high dose of ibuprofin to counter act adult stem cell to reduce any ill effects receiving stem cells from another person.

I apologize if i am incorrect, but like i said, i am out of touch with research...and i am much more happier now too. I've decided to leave research up to the professionals and let myself be to Micah what none of them can...a father without worrying about saving thousands of children/young adults. i like my job better.
We had to do the same thing. At some point we had to stop looking and reading every new piece of research info that came out and just be.......it would take our hopes sky high to only have them come crashing down again. For our family, that emoitional rollercoaster was too much to handle. Life is good for us this way!

MicahsDaddy said:
Char,

Yes, each exon will be tested. But my understanding is that as these first groups show positive results, then the process will speed up. At the current rate, it would take approx 400 +/- years to test each one. Obviously, there's no time for that. So, after the first few show positive results, then there would be multiple studies going on simultaneously with different exons. And, because of previous positive results, drug to market should be expedited because of those successes.

But, for exon skipping, i don't count on it to work for every child. There are plenty of cases of BMD that progress as rapid as DMD.

That's why my interest is in a cocktail of drugs.

As for Italy, I believe you are talking about Dr. Cossu. His study was some kind of meso(or immuno)-angioblast or something. I have been out of touch with research for quite sometime now, so my recollection could be incorrect. Anyway, if i remember correctly, it was basically a super high dose of ibuprofin to counter act adult stem cell to reduce any ill effects receiving stem cells from another person.

I apologize if i am incorrect, but like i said, i am out of touch with research...and i am much more happier now too. I've decided to leave research up to the professionals and let myself be to Micah what none of them can...a father without worrying about saving thousands of children/young adults. i like my job better.

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