Are you hopeful?

[tammykrumrey]

My daughters' CF doctor is VERY excited about the VX-770. This is what he had to say about it during clinic two weeks ago:
As the state it is in now, VX-770 will not help delF508, but there are other drugs in the pipeline that could POSSIBLY be used in conjunction with the VX-770, that holds the potential for helping those with delF508.
He is really a hands on doctor, he spends half of his time in a lab and the rest of his time seeing patients. He said that he has never really been excited about a drug as he is with this one, simply for the fact that it changed the sweat test results of all the patients who were taking it from the 90s down to the 50s. He said that this has never happened before and is a very exciting breakthrough. The other statistics the study shows, such as improvements of PFTs and Nasal differentials (spelling?) can vary slightly from day to day, so he doesn't really get overly excited when studies come out using those type of statistics alone.
He told me that it is possible that in the next year to two, he could be asking Kayla and Hannah to participate in clinical studies/trials (they carry delF508 and 1898+1G>A.)

I am hopeful that there will be great changes to come, but not a cure to say. But I do feel that I am blessed that my girls were born when they were, and will be healthy enough to benefit from what may come down the pipeline.

That is my ultimate goal...keep them as healthy as possible because I want them to benefit from each great stride that is taken!

 

[tammykrumrey]

My daughters' CF doctor is VERY excited about the VX-770. This is what he had to say about it during clinic two weeks ago:
As the state it is in now, VX-770 will not help delF508, but there are other drugs in the pipeline that could POSSIBLY be used in conjunction with the VX-770, that holds the potential for helping those with delF508.
He is really a hands on doctor, he spends half of his time in a lab and the rest of his time seeing patients. He said that he has never really been excited about a drug as he is with this one, simply for the fact that it changed the sweat test results of all the patients who were taking it from the 90s down to the 50s. He said that this has never happened before and is a very exciting breakthrough. The other statistics the study shows, such as improvements of PFTs and Nasal differentials (spelling?) can vary slightly from day to day, so he doesn't really get overly excited when studies come out using those type of statistics alone.
He told me that it is possible that in the next year to two, he could be asking Kayla and Hannah to participate in clinical studies/trials (they carry delF508 and 1898+1G>A.)

I am hopeful that there will be great changes to come, but not a cure to say. But I do feel that I am blessed that my girls were born when they were, and will be healthy enough to benefit from what may come down the pipeline.

That is my ultimate goal...keep them as healthy as possible because I want them to benefit from each great stride that is taken!

 

[tammykrumrey]

My daughters' CF doctor is VERY excited about the VX-770. This is what he had to say about it during clinic two weeks ago:
As the state it is in now, VX-770 will not help delF508, but there are other drugs in the pipeline that could POSSIBLY be used in conjunction with the VX-770, that holds the potential for helping those with delF508.
He is really a hands on doctor, he spends half of his time in a lab and the rest of his time seeing patients. He said that he has never really been excited about a drug as he is with this one, simply for the fact that it changed the sweat test results of all the patients who were taking it from the 90s down to the 50s. He said that this has never happened before and is a very exciting breakthrough. The other statistics the study shows, such as improvements of PFTs and Nasal differentials (spelling?) can vary slightly from day to day, so he doesn't really get overly excited when studies come out using those type of statistics alone.
He told me that it is possible that in the next year to two, he could be asking Kayla and Hannah to participate in clinical studies/trials (they carry delF508 and 1898+1G>A.)

I am hopeful that there will be great changes to come, but not a cure to say. But I do feel that I am blessed that my girls were born when they were, and will be healthy enough to benefit from what may come down the pipeline.

That is my ultimate goal...keep them as healthy as possible because I want them to benefit from each great stride that is taken!

 

[tammykrumrey]

My daughters' CF doctor is VERY excited about the VX-770. This is what he had to say about it during clinic two weeks ago:
As the state it is in now, VX-770 will not help delF508, but there are other drugs in the pipeline that could POSSIBLY be used in conjunction with the VX-770, that holds the potential for helping those with delF508.
He is really a hands on doctor, he spends half of his time in a lab and the rest of his time seeing patients. He said that he has never really been excited about a drug as he is with this one, simply for the fact that it changed the sweat test results of all the patients who were taking it from the 90s down to the 50s. He said that this has never happened before and is a very exciting breakthrough. The other statistics the study shows, such as improvements of PFTs and Nasal differentials (spelling?) can vary slightly from day to day, so he doesn't really get overly excited when studies come out using those type of statistics alone.
He told me that it is possible that in the next year to two, he could be asking Kayla and Hannah to participate in clinical studies/trials (they carry delF508 and 1898+1G>A.)

I am hopeful that there will be great changes to come, but not a cure to say. But I do feel that I am blessed that my girls were born when they were, and will be healthy enough to benefit from what may come down the pipeline.

That is my ultimate goal...keep them as healthy as possible because I want them to benefit from each great stride that is taken!

 

[tammykrumrey]

My daughters' CF doctor is VERY excited about the VX-770. This is what he had to say about it during clinic two weeks ago:
<br />As the state it is in now, VX-770 will not help delF508, but there are other drugs in the pipeline that could POSSIBLY be used in conjunction with the VX-770, that holds the potential for helping those with delF508.
<br />He is really a hands on doctor, he spends half of his time in a lab and the rest of his time seeing patients. He said that he has never really been excited about a drug as he is with this one, simply for the fact that it changed the sweat test results of all the patients who were taking it from the 90s down to the 50s. He said that this has never happened before and is a very exciting breakthrough. The other statistics the study shows, such as improvements of PFTs and Nasal differentials (spelling?) can vary slightly from day to day, so he doesn't really get overly excited when studies come out using those type of statistics alone.
<br />He told me that it is possible that in the next year to two, he could be asking Kayla and Hannah to participate in clinical studies/trials (they carry delF508 and 1898+1G>A.)
<br />
<br />I am hopeful that there will be great changes to come, but not a cure to say. But I do feel that I am blessed that my girls were born when they were, and will be healthy enough to benefit from what may come down the pipeline.
<br />
<br />That is my ultimate goal...keep them as healthy as possible because I want them to benefit from each great stride that is taken!

 

[zeeannie]

I don't think it's silly to be optimistic about a cure in our kids' lifetimes. When you look at the progress being made in the amount of time it has happened it seems likely. The protein therapies and gene therapies are particularly exciting as they know that they can correct the protein or the gene, they just need to figure out how to deliver the appropriate amount in an appropriate form. Meanwhile, our kids still do have CF and no matter how close the cure is, as long as my kid still has it and requires therapy I will never feel like slacking on her treatments. If a cure does become available, we want our kids to be in the best possible shape they can be when it happens. A cure won't reverse lung damage that has already occurred, or take away CFRD. So I will be as hopeful as I can be while still dealing with the day to day realities of taking care of a child with CF. That's one thing you can say about a CF parent, we are pragmatic in the face of everything!

 

[zeeannie]

I don't think it's silly to be optimistic about a cure in our kids' lifetimes. When you look at the progress being made in the amount of time it has happened it seems likely. The protein therapies and gene therapies are particularly exciting as they know that they can correct the protein or the gene, they just need to figure out how to deliver the appropriate amount in an appropriate form. Meanwhile, our kids still do have CF and no matter how close the cure is, as long as my kid still has it and requires therapy I will never feel like slacking on her treatments. If a cure does become available, we want our kids to be in the best possible shape they can be when it happens. A cure won't reverse lung damage that has already occurred, or take away CFRD. So I will be as hopeful as I can be while still dealing with the day to day realities of taking care of a child with CF. That's one thing you can say about a CF parent, we are pragmatic in the face of everything!

 

[zeeannie]

I don't think it's silly to be optimistic about a cure in our kids' lifetimes. When you look at the progress being made in the amount of time it has happened it seems likely. The protein therapies and gene therapies are particularly exciting as they know that they can correct the protein or the gene, they just need to figure out how to deliver the appropriate amount in an appropriate form. Meanwhile, our kids still do have CF and no matter how close the cure is, as long as my kid still has it and requires therapy I will never feel like slacking on her treatments. If a cure does become available, we want our kids to be in the best possible shape they can be when it happens. A cure won't reverse lung damage that has already occurred, or take away CFRD. So I will be as hopeful as I can be while still dealing with the day to day realities of taking care of a child with CF. That's one thing you can say about a CF parent, we are pragmatic in the face of everything!

 

[zeeannie]

I don't think it's silly to be optimistic about a cure in our kids' lifetimes. When you look at the progress being made in the amount of time it has happened it seems likely. The protein therapies and gene therapies are particularly exciting as they know that they can correct the protein or the gene, they just need to figure out how to deliver the appropriate amount in an appropriate form. Meanwhile, our kids still do have CF and no matter how close the cure is, as long as my kid still has it and requires therapy I will never feel like slacking on her treatments. If a cure does become available, we want our kids to be in the best possible shape they can be when it happens. A cure won't reverse lung damage that has already occurred, or take away CFRD. So I will be as hopeful as I can be while still dealing with the day to day realities of taking care of a child with CF. That's one thing you can say about a CF parent, we are pragmatic in the face of everything!

 

[zeeannie]

I don't think it's silly to be optimistic about a cure in our kids' lifetimes. When you look at the progress being made in the amount of time it has happened it seems likely. The protein therapies and gene therapies are particularly exciting as they know that they can correct the protein or the gene, they just need to figure out how to deliver the appropriate amount in an appropriate form. Meanwhile, our kids still do have CF and no matter how close the cure is, as long as my kid still has it and requires therapy I will never feel like slacking on her treatments. If a cure does become available, we want our kids to be in the best possible shape they can be when it happens. A cure won't reverse lung damage that has already occurred, or take away CFRD. So I will be as hopeful as I can be while still dealing with the day to day realities of taking care of a child with CF. That's one thing you can say about a CF parent, we are pragmatic in the face of everything!

 

[mom4holly]

I like to think I am optimistic but realistically I don't think there will be a CURE in Holly's lifetime. Do I think that there will be treatment to manage it better...YES, do I think it's gonna get easier...YES but I still think that it might because something that there are only meds to control it & stop it from causing an untimely death instead of a cure. This past few weeks I met with 2 of the people from the cff for lunch & while i was in tears when he told me about vertex or vx-770 I had to remind myself that I can't let those things get me too excited because I don't want to be let down.

 

[mom4holly]

I like to think I am optimistic but realistically I don't think there will be a CURE in Holly's lifetime. Do I think that there will be treatment to manage it better...YES, do I think it's gonna get easier...YES but I still think that it might because something that there are only meds to control it & stop it from causing an untimely death instead of a cure. This past few weeks I met with 2 of the people from the cff for lunch & while i was in tears when he told me about vertex or vx-770 I had to remind myself that I can't let those things get me too excited because I don't want to be let down.

 

[mom4holly]

I like to think I am optimistic but realistically I don't think there will be a CURE in Holly's lifetime. Do I think that there will be treatment to manage it better...YES, do I think it's gonna get easier...YES but I still think that it might because something that there are only meds to control it & stop it from causing an untimely death instead of a cure. This past few weeks I met with 2 of the people from the cff for lunch & while i was in tears when he told me about vertex or vx-770 I had to remind myself that I can't let those things get me too excited because I don't want to be let down.

 

[mom4holly]

I like to think I am optimistic but realistically I don't think there will be a CURE in Holly's lifetime. Do I think that there will be treatment to manage it better...YES, do I think it's gonna get easier...YES but I still think that it might because something that there are only meds to control it & stop it from causing an untimely death instead of a cure. This past few weeks I met with 2 of the people from the cff for lunch & while i was in tears when he told me about vertex or vx-770 I had to remind myself that I can't let those things get me too excited because I don't want to be let down.

 

[mom4holly]

I like to think I am optimistic but realistically I don't think there will be a CURE in Holly's lifetime. Do I think that there will be treatment to manage it better...YES, do I think it's gonna get easier...YES but I still think that it might because something that there are only meds to control it & stop it from causing an untimely death instead of a cure. This past few weeks I met with 2 of the people from the cff for lunch & while i was in tears when he told me about vertex or vx-770 I had to remind myself that I can't let those things get me too excited because I don't want to be let down.

 

[westonsmom]

Maybe it is denial or just that I need something to hold on to, but YES in my heart I know there will be a cure in Weston's lifetime. Does that sound bold and maybe uneducated. Maybe. Why else would I be fundraising my a** off to find one if I didn't believe there would ever be one. My son WILL live far into adulthood. CF is a demon but I have been equipped with a sharp sword to fight it away from my son.

 

[westonsmom]

Maybe it is denial or just that I need something to hold on to, but YES in my heart I know there will be a cure in Weston's lifetime. Does that sound bold and maybe uneducated. Maybe. Why else would I be fundraising my a** off to find one if I didn't believe there would ever be one. My son WILL live far into adulthood. CF is a demon but I have been equipped with a sharp sword to fight it away from my son.

 

[westonsmom]

Maybe it is denial or just that I need something to hold on to, but YES in my heart I know there will be a cure in Weston's lifetime. Does that sound bold and maybe uneducated. Maybe. Why else would I be fundraising my a** off to find one if I didn't believe there would ever be one. My son WILL live far into adulthood. CF is a demon but I have been equipped with a sharp sword to fight it away from my son.

 

[westonsmom]

Maybe it is denial or just that I need something to hold on to, but YES in my heart I know there will be a cure in Weston's lifetime. Does that sound bold and maybe uneducated. Maybe. Why else would I be fundraising my a** off to find one if I didn't believe there would ever be one. My son WILL live far into adulthood. CF is a demon but I have been equipped with a sharp sword to fight it away from my son.

 

[westonsmom]

Maybe it is denial or just that I need something to hold on to, but YES in my heart I know there will be a cure in Weston's lifetime. Does that sound bold and maybe uneducated. Maybe. Why else would I be fundraising my a** off to find one if I didn't believe there would ever be one. My son WILL live far into adulthood. CF is a demon but I have been equipped with a sharp sword to fight it away from my son.