bigbird 60,493 Posted December 24, 2021 Share Posted December 24, 2021 ...but I know many have interest in and have graciously prayed for us. 2 6 Link to comment Share on other sites More sharing options...
Popular Post bigbird 60,493 Posted December 24, 2021 Author Popular Post Share Posted December 24, 2021 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! 5 20 Link to comment Share on other sites More sharing options...
wdefromtx 3,159 Posted December 24, 2021 Share Posted December 24, 2021 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. 1 Link to comment Share on other sites More sharing options...
AUDub 11,147 Posted December 24, 2021 Share Posted December 24, 2021 Sounds promising! I hope the trial goes well! 1 Link to comment Share on other sites More sharing options...
alexava 6,973 Posted December 24, 2021 Share Posted December 24, 2021 This is going to be the ticket my friend! I can feel it! Keep us posted! 1 Link to comment Share on other sites More sharing options...
I_M4_AU 7,793 Posted December 24, 2021 Share Posted December 24, 2021 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. 1 Link to comment Share on other sites More sharing options...
Grumps 3,704 Posted December 24, 2021 Share Posted December 24, 2021 Praise God and Merry Christmas!!! James and you and your family are due for some awesome news! 1 Link to comment Share on other sites More sharing options...
SaltyTiger 7,789 Posted December 24, 2021 Share Posted December 24, 2021 Merry Christmas Great news on hope! Keep the faith bird 1 Link to comment Share on other sites More sharing options...
TitanTiger 20,472 Posted December 25, 2021 Share Posted December 25, 2021 That’s great news! 1 Link to comment Share on other sites More sharing options...
augolf1716 21,103 Posted December 25, 2021 Share Posted December 25, 2021 Wow so great to hear 1 Link to comment Share on other sites More sharing options...
McLoofus 35,182 Posted December 26, 2021 Share Posted December 26, 2021 Hell yeah dude!!!! Hope is hope!!!! I'm sure this is at least somewhat a result of a lot of diligence on the part of you and Mrs. Bird so well done and woo hoo! 1 Link to comment Share on other sites More sharing options...
homersapien 11,362 Posted December 27, 2021 Share Posted December 27, 2021 (edited) 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 December 27, 2021 by homersapien Link to comment Share on other sites More sharing options...
bigbird 60,493 Posted December 27, 2021 Author Share Posted December 27, 2021 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? Link to comment Share on other sites More sharing options...
homersapien 11,362 Posted December 27, 2021 Share Posted December 27, 2021 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.) 1 Link to comment Share on other sites More sharing options...
bigbird 60,493 Posted December 27, 2021 Author Share Posted December 27, 2021 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. 1 Link to comment Share on other sites More sharing options...
AU9377 4,924 Posted December 27, 2021 Share Posted December 27, 2021 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! 1 Link to comment Share on other sites More sharing options...
cbo 7,835 Posted December 29, 2021 Share Posted December 29, 2021 This made a terrible day much better. Hoping for the best, BB. You and yours definitely deserve it. 1 Link to comment Share on other sites More sharing options...
aubiefifty 16,755 Posted December 31, 2021 Share Posted December 31, 2021 "Really not sure where to put this..." ask golf i bet he will tell ya? lol Link to comment Share on other sites More sharing options...
Popular Post bigbird 60,493 Posted September 3, 2022 Author Popular Post Share Posted September 3, 2022 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! 1 15 Link to comment Share on other sites More sharing options...
Ausburn2 487 Posted September 3, 2022 Share Posted September 3, 2022 Blessings ahead for your family. 1 Link to comment Share on other sites More sharing options...
SaltyTiger 7,789 Posted September 3, 2022 Share Posted September 3, 2022 So happy for your excitement. Best to your family. 1 Link to comment Share on other sites More sharing options...
I_M4_AU 7,793 Posted September 3, 2022 Share Posted September 3, 2022 Great news and just in time. Is James home today? Link to comment Share on other sites More sharing options...
bigbird 60,493 Posted September 3, 2022 Author Share Posted September 3, 2022 3 minutes ago, I_M4_AU said: Great news and just in time. Is James home today? No, probably Tuesday 1 Link to comment Share on other sites More sharing options...
Leftfield 2,593 Posted September 3, 2022 Share Posted September 3, 2022 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? Link to comment Share on other sites More sharing options...
bigbird 60,493 Posted September 3, 2022 Author Share Posted September 3, 2022 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. 1 Link to comment Share on other sites More sharing options...
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