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So how can something like this be brought to trial? Perhaps someone at PPMD can comment?

Hi Amit - PPMD, along with United Parent Project MD, is co-funding studies necessary to verify these initial results and lay the groundwork for a human clinical trial. This project is part of our “Now” strategy to test approved drugs.

Posted 5/20/2012: Study FAQ Sheet - DuchenneConnect

thank you for the information!

Perhaps, Tamoxifen can be tested older DMD patients first where the risk is lower.

PPMD:  The FAQ link says the goal was to have a clinical trial by the end of 2012 or early 2013. 

It's early 2013.  

If this worked in a human trial, this is an ideal drug.  FDA approved, so it'd be available off-label immediately (with supporting data from a clnical trial).  And most importantly, this drug is a reasonable cost to purchase out of pocket, which is what you'd need to do off-label.

By supporting a researcher in Geneva - does that mean any clinical trial would likely not take place in the US?   

Dear Andrea, 

Thank you for your message and for posting this, excuse me for posting my dilema but I wanted to know if more parents feel the same way, somehow I wanted to rely on every other parent struggling through the same feeling.

I stay clear and positive as always, but I do believe that trials of FDA approved drugs should be a priority if the drug results to be a hot shot even if it is in animal studies. We need someone to tell us a possible dose for kids, we have all made somehow our own trials sometimes trying different doses, on and off, daily, switching to more or less of this and that, we need a guide on something as good as this. (based on the article). 

I won´t try it freely on my son but some medical advice and FASTER "trial" to find a dose wich reduces risk of bad side effects is what I ask for, then we could all at least try, remember it´s FDA approved and we have years of investigation and trials, lets have a conclusion about this data having in mind what it can do to muscles.

Once again thank you Andrea,

Best for you and your family.

JP.

Andrea Cleary said:

Dear Juan,

I am very sorry for the predicament you find yourself in. I just posted this as a piece of information only, but remember it is just a mouse study! I just think that any therapy that shows some real, demonstrated promise should be further explored for all our boy's sakes. None of us want to expose our children to more risks than benefits, but that is a part of research. Each and every medication can have bad side-effects (including streroids), and we all react to drugs differently. It's an idividual and family decision, a choice that we all have to make, hopefully sooner than later. I personally rather have the opportunity to make a decision than to have no options at all. The Tamoxifen study is just too early to say one way or the other if it could be a potential therapy. In an ideal world, there would be no Duchenne, no medication side-effects, and no animal testing, but we are not there yet.

Let's try to stay focused on the positive. Lord knows it's not always easy. Take care of you and yours, Juan.

A.

We agree Juan!
 

PPMD is already funding this work to do the next steps for a human study--we need to understand dosing better and we are looking at tamoxifen in combination with prednisone.  All drugs do have side effects but we might be able to adjust the dosing to minimize those side effects.  Those are the kinds of things that we are trying to understand before starting a human clinical study.  We will post updates as they are avaialble.

 

Best Wishes,

Sharon (PPMD VP Research0

Thanks for the updates, Sharon.

A.

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