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bigbird
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This is awesome news!! Best Christmas present your family could get!! 
 

Prayers for him and that this drug will work for him and that his body responds to it. 

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Well, Merry Christmas and a Happy and Healthy New Year.  What great news, if we don’t have hope we can’t survive. Congrats on the trial.

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On 12/24/2021 at 11:02 AM, bigbird said:

We have heard about the potential of this drug a few years ago, but we're never able to get any traction with our attempts to be involved.

As of the end of last week, after 5 years of trying, Novo has granted James a compassionate use exception to join the phase III trial!!!!

We don't know if it will work or not for him (he is one of 15 worldwide with his exact condition/mutation/severity/inhibitors) but this is the first time in 15 years we have some hope for a better life for him.

I just needed to share.

 

Praise God! 

Merry Christmas!

That's great news!!  (I was about to ask if you have tried to get into a trial.) 

Do you know about how many people this condition affects?

Edited by homersapien
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14 minutes ago, homersapien said:

Do you know about how many people this condition affects?

Hemophilia in general, Hemophilia A, Hemophilia B, or those with inhibitors?

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2 minutes ago, bigbird said:

Hemophilia in general, Hemophilia A, Hemophilia B, or those with inhibitors?

I am generally ignorant about hemophilia, but I was referring to whatever group this particular drug was designed to help.

(The reason I asked was the lack of commercial incentives for drug companies to develop therapies for "orphan" or rare diseases.)

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1 minute ago, homersapien said:

I am generally ignorant about hemophilia, but I was referring to whatever group this particular drug was designed to help.

(The reason I asked was the lack of commercial incentives for drug companies to develop therapies for "orphan" or rare diseases.)

Here are some links, I'm more than happy to answer/educate any questions

https://www.ajmc.com/view/prevalence-of-hemophilia-worldwide-is-triple-that-of-previous-estimates-new-study-says-

https://www.cdc.gov/ncbddd/hemophilia/inhibitors.html#:~:text=Approximately 1 in 5 people,or prevent their bleeding episodes.

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On 12/24/2021 at 11:02 AM, bigbird said:

We have heard about the potential of this drug a few years ago, but we're never able to get any traction with our attempts to be involved.

As of the end of last week, after 5 years of trying, Novo has granted James a compassionate use exception to join the phase III trial!!!!

We don't know if it will work or not for him (he is one of 15 worldwide with his exact condition/mutation/severity/inhibitors) but this is the first time in 15 years we have some hope for a better life for him.

I just needed to share.

 

Praise God! 

Merry Christmas!

My family has also dealt with this issue.  My mother lost a brother in the 1960s and another in the 80s that both dealt with Hemophilia. Her brother that died in the 80s did not die as a result of his Hemophilia, but it contributed to the condition that eventually took his life.  I was lucky. It was a 50/50 roll of the dice when I was born.  This is great news.

As you know, the good news is that this doesn't prevent people from living full lives today as it once did, but it is a burden nonetheless and makes dealing with every day medical issues much more complicated at times.  Very glad that James was accepted into this trial.  Good luck and prayers for good results!

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"Really not sure where to put this..."       ask golf i bet he will tell ya?  lol

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  • 8 months later...
3 minutes ago, I_M4_AU said:

Great news and just in time.  Is James home today?

No, probably Tuesday 

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9 hours ago, bigbird said:

Well, it's here.

After almost a year of negotiations, contracts, new protocols, and multiple hospital committee reviews and approvals we had the final site visit last week from Novo nordisk and are scheduled to have our first appointment and medication on September 13th.   

We've been discussing this drug for 9 years and it's finally happening. We are beyond ready and excited. Any and all continued prayers or well wishes are appreciated. Thanks, Guys!

Will be thinking of James, you, and your family, Bird. 

Has Novo Nordisk said how long they expect the trial to run, both for James and overall? Any idea how quickly a response is expected?

 

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1 hour ago, Leftfield said:

Will be thinking of James, you, and your family, Bird. 

Has Novo Nordisk said how long they expect the trial to run, both for James and overall? Any idea how quickly a response is expected?

 

Our initial is 6 months at which time we can opt out or stay in for an additional 6 months. After that, we'll reassess and make a determination of efficacy and if worth the cost.  

Good question.  Because his condition is completely random, I don't know how quickly we'll see anything. Basically whenever he has his first bleed after we start. I'd assume, with his pattern of bleeds, maybe 2-4 days from the initial.

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