Diabetes drug in phase 2 clinical trial Hope for DMD

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Thank you Jason, you and your brother gives me hope for my son who is 6 and his name is Jacob.

You welcome, glad to help 

Same issue we were talking about in another thread.

"We hope that these exciting findings will serve as the basis for future clinical trials within the next five to 10 years," Professor Lynch said.

Future trials in five to ten years!?!  What takes so long?  

I agree Keith, lets keep fighting for quicker response time after all our boys mostly live day by day and not year by year.

Keith Van Houten said:

Same issue we were talking about in another thread.

"We hope that these exciting findings will serve as the basis for future clinical trials within the next five to 10 years," Professor Lynch said.

Future trials in five to ten years!?!  What takes so long?  

I sent this article to Sharon Hesterlee, ask her to look in to the research. Maybe Parent Project can assist the company in getting on track for a Duchenne clinical trial with Orphan drug status.  Hopefully, if approved for diabetes it can speed things up for DMD

I received an e-mailed from Sharon Hesterlee. She sent the article to Lee Sweeney PPMD's Scientific Advisory Committee Chair to see what he thinks about it. 

Interesting how many links Duchenne has to Diabetes, even before the steroids. Makes me think a low glycemic index diet is the way to go. If only mine didn't love carbs so much!

 

http://www.ncbi.nlm.nih.gov/pubmed/3965491

Some where deep inside my mind I think the cure is right in front of us, but I wish whole heartedly we could find it.

You are right, Thomas ! It's so near but so far.

Rupjani

Thomas Hilliker said:

Some where deep inside my mind I think the cure is right in front of us, but I wish whole heartedly we could find it.

I really think that with combining two or three drugs the drugs in the pipeline we are there. A lot of boys going into wheelchairs today will have a normal life spans(and walk with robotic exoskeletons).   And DMD boys born today will never loose that ability to walk. The next 3 to 5 years are going to very exciting to watch and benefit from. when I was born in 1979 the Dystrophin gene was not discovered there has been huge progress in my 32 years with DMD. This is best time to be unlucky enough to have DMD.

Rupjani B said:

You are right, Thomas ! It's so near but so far.

Rupjani

Thomas Hilliker said:

Some where deep inside my mind I think the cure is right in front of us, but I wish whole heartedly we could find it.

Jason, I really appreciate the information you post. Your comment here pretty much outlines the cautious hope I have for my own son.  You give us a lot to think about and a lot to hope for. Thank you.


Jason Darienzo said:

I really think that with combining two or three drugs the drugs in the pipeline we are there. A lot of boys going into wheelchairs today will have a normal life spans(and walk with robotic exoskeletons).   And DMD boys born today will never loose that ability to walk. The next 3 to 5 years are going to very exciting to watch and benefit from. when I was born in 1979 the Dystrophin gene was not discovered there has been huge progress in my 32 years with DMD. This is best time to be unlucky enough to have DMD.

Rupjani B said:

You are right, Thomas ! It's so near but so far.

Rupjani

Thomas Hilliker said:

Some where deep inside my mind I think the cure is right in front of us, but I wish whole heartedly we could find it.

Jason, when I look into my sons eyes I see a beautiful boy that wants to grow up and be his own person.  I know you have beat most of the odds and lived a long time with Duchene and that gives not only me but a lot of people hope that our boys will make it that long.  Whenever I think of a cure, I think of all the people who have been affected by this disease and thank them for the knowledge that we have today.  Without them which includes you we may not have known what we know today.  You know what I want Jason; I want a cure that would not only cure DMD boys but restore them with all their glory.  It hurts so much to talk about this but yet it helps so much and for that I wish to extend a personal thank you to you. Hang in there Jason when this cure comes it will come fast and we will hopefully be able to put all this DMD stuff behind us for good.  I am personally looking forward to when that day comes even if it doesn’t come in time for my own son who is only six.  My daughter told me the other day that she wanted to be a geneticist when she grows up.  Katie is only 12 but I have high hopes that she will.  She says that if a cure is not found by then she will find one and name it Buddies Cure after our beautiful son Jacob.  What my daughter told me was so awesome that I broke down and cried right in front of her, something I have never done before.  At that moment I knew that if a 12 year old could say something like that there must be thousands of people with her same passion to solve this disease.  I apologize if I keep rambling but I don’t know where to stop or even if I can.  I know words aren’t going to cure DMD but if they could I would never stop talking.

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