Why isn't Duchenne being given same consideration as AIDS?

    Duchenne Muscular Dystrophy needs treatments immediately. In that context, consider this: In early 1984, HIV was discovered as the cause of AIDS. In March 1987 AZT was approved by the FDA.. That is Accelerated Approval. Dystrophin was discovered as the cause of Duchenne in 1987. 27 years later Duchenne can not get one drug. AZT didn't cure AIDS and they barely knew anything about HIV. AIDS desperately needed something, we desperately need something to start raising Dystrophin levels.

    The goal of HIV/AIDS drugs is too reduce the levels of the HIV in the blood.  AZT was not the cure and they needed more drugs to continue to reduce levels.  Every little gain in reducing the level of Virus was approved as another drug.  This just like raising Dystrophin levels.  Ultimately restoration of Dystrophin levels is the road to a cure.  Every drug that can raise levels we need.

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Jason, what are your thoughts on how we can jump-start things, beyond what we have done so far?

I'm thinking radical changes need to be made, without "starting over", which will waste more time. Is it the genral public we need to work on, is it getting celebrities on board?

Everyone, throw your ideas out here so we can get some discussions going, please.

A.

           Andrea i think i have confused you just a bit.   Im Richard, Jasons brother.  Me and Jason both post so im sorry we did not clear that up sooner.    The change i think needs to be how the regulatory authorities view the treatment path for Duchenne.  We know lack of Dystrophin is the cause.   I think we have to make it clear that we need any treatment that raises dystrophin levels.  In the case of AIDS  multiple drugs are used to lower virus levels.  So there is no requirements that it must be one treatment alone that raises dystrophin high enough.  Any medicine that raises dystrophin levels safely should be approved.          

    One next step is to make sure the FDA and everyone is aware of the AIDS analogy.   Then ask why should  Duchenne be treated any differently?  

    The other issue is how do we have the FDA make the right decision.   Yes we can argue with them about results and safety.   However, they feel they are the authority so that is limited.   I think the time for discussion with the FDA is over.   Political pressure is the only real way to force the FDA's hand.   Having Congressmen and Senators asking the FDA to move faster is great.  The most important is constant political pressure.    

Sorry, I did it again! But I was sure I saw the name Jason. Again, please forgive me Richard.

I do like the comparison to AIDS, I think that would have an impact if we could get that into people's skulls. 

As to the political pressure, how can we do that in an innovative and quick manner? I just think that emails and letters can so easily go unread or deleted. We need something memorable and in their face, without turning them off to our voice.

     Oh no need to apologize Andrea.   Your a huge supporter of my Robotic Exoskeleton for Duchenne facebook group so im big fan of yours.   Well as far as political pressure,  Your aware that The head of the FDA, Margaet Hamburg,  was asked two times in the last week about Eteplirsen.   A Congressman, a Senator and a news reporter asked her.  Charley's Fund and others got that done.

     I agree with you letters help but how much can a letter really do.  However,  we can't stop writing.  Persistence can pay off.   I hope the FDA is getting frustrated by our letters.

     Your right Muscular Dystrophy needs high profile support again.  Who and how i just am not sure.   I mean even the celebrities that did the telethon dont seem to want to take it on.

      Maybe this is crazy but if the FDA gives a flat out no why not go elswhere.  With the U.S. and Russia in this struggle im sure Russia would want to help.  Yes that is a controversial idea but we need this drug now.

 

Why did Obama find time to comment on a botched execution of a _______ criminal suggesting it was a racial thing and not find time to comment on THE RACE TO YES petition.  Seems his priorities are almost criminal in nature! Maybe we need knew head of FDA with no economic ties!  Read about her recent actions with her former employer and the conflicts of her husband Peter Brown! 

Read the NORD report "Quantum of Effectiveness"   Todays FDA should be deemed the Quantum of Ineffectiveness!!!!!

Criminal?  Really.  

Remind me of who it was that signed FDASIA into law, with enhancements and clarification of the accelerated approval pathway?  Oh yeah, it was Obama.

And whose signature legislation was it that will keep boys from being excluded from insurance coverage because they're sick, or maxing out their benefits - so they can actually get the drug?  Oh yeah.  Obama.

 



micro kid said:

Why did Obama find time to comment on a botched execution of a _______ criminal suggesting it was a racial thing and not find time to comment on THE RACE TO YES petition.  Seems his priorities are almost criminal in nature! Maybe we need knew head of FDA with no economic ties!  Read about her recent actions with her former employer and the conflicts of her husband Peter Brown! 

Read the NORD report "Quantum of Effectiveness"   Todays FDA should be deemed the Quantum of Ineffectiveness!!!!!

Sorry I offended you.  Please observe I used the qualifier "almost".  The fact that he has in the past done good things does not excuse him from in the present from continuing to do good things!  The president made a commitment for the White House to answer petitions with 100 thousand or more signatures.  There are a number of petitions now unanswered.

Jon Moulton pointed out  "His comment was in response to a reporter's question."  I am sorry I did not catch that fact. 

I will point out that the FDA has been more aggressive in the past on rare diseases as witnessed in the "Quantum of Effectiveness" document, as they also were with AIDs.

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