Treatments based on genetics

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tammykrumrey

Guest
My girls had CF clinic last week, and they were discussing the fact that in the near future they would be tayloring our (their) treatments based on genetic mutations. I was also asked to fill out a survey regarding my feelings about it, in which I did fill out. Of course, I would have more questions regarding treatments before agreeing to anything.

Has anyone else heard this from their child's clinic?
 
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tammykrumrey

Guest
My girls had CF clinic last week, and they were discussing the fact that in the near future they would be tayloring our (their) treatments based on genetic mutations. I was also asked to fill out a survey regarding my feelings about it, in which I did fill out. Of course, I would have more questions regarding treatments before agreeing to anything.

Has anyone else heard this from their child's clinic?
 
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tammykrumrey

Guest
My girls had CF clinic last week, and they were discussing the fact that in the near future they would be tayloring our (their) treatments based on genetic mutations. I was also asked to fill out a survey regarding my feelings about it, in which I did fill out. Of course, I would have more questions regarding treatments before agreeing to anything.

Has anyone else heard this from their child's clinic?
 

angelsmom

New member
We were just at our CF clinic last week too and nothing was said about this.

I'm curious . . . what clinic do you go to?
 

angelsmom

New member
We were just at our CF clinic last week too and nothing was said about this.

I'm curious . . . what clinic do you go to?
 

angelsmom

New member
We were just at our CF clinic last week too and nothing was said about this.

I'm curious . . . what clinic do you go to?
 
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tammykrumrey

Guest
We go to St. Louis Children's Hospital's CF clinic. It was mentioned for a brief minute while in clinic. Then it was mentioned again during a Parent Advisory Board that I volunteer to sit on. It was not discussed in any detail, so I was just curious if anyone else has had their clinic mention it. I guess in time we'll learn more<img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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tammykrumrey

Guest
We go to St. Louis Children's Hospital's CF clinic. It was mentioned for a brief minute while in clinic. Then it was mentioned again during a Parent Advisory Board that I volunteer to sit on. It was not discussed in any detail, so I was just curious if anyone else has had their clinic mention it. I guess in time we'll learn more<img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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tammykrumrey

Guest
We go to St. Louis Children's Hospital's CF clinic. It was mentioned for a brief minute while in clinic. Then it was mentioned again during a Parent Advisory Board that I volunteer to sit on. It was not discussed in any detail, so I was just curious if anyone else has had their clinic mention it. I guess in time we'll learn more<img src="i/expressions/face-icon-small-smile.gif" border="0">
 

Rebjane

Super Moderator
I think this is very interesting. When my daughter was first diagnosed with CF over 4 years ago, a CF doctor said to us that someday in the future this would happen. That the particular mutation/ or mutations you have would be treated specifically and a Cf'er may be on one drug for each particular mutation. For example, my daughter has delta F08 and W1282X. So she could potentially be treated with drugs to target channels that open the sodium chloride channels in each of those particular pathways/mutations. He said you would not need the function to be 100% to help a Cf'er . This doc could explain this so much better than me right now. Anyway, the drug PCT124 is a particular therapy that works on nonsense mutations such as W1282X that is in clinical trials that is targeted to turn on a pathway to make these sodium/chloride channels work better. I have not heard about this specifically in our clinic we go to now. But it's great they are trying to get data from you. Hopefully this would be treatment that works.
 

Rebjane

Super Moderator
I think this is very interesting. When my daughter was first diagnosed with CF over 4 years ago, a CF doctor said to us that someday in the future this would happen. That the particular mutation/ or mutations you have would be treated specifically and a Cf'er may be on one drug for each particular mutation. For example, my daughter has delta F08 and W1282X. So she could potentially be treated with drugs to target channels that open the sodium chloride channels in each of those particular pathways/mutations. He said you would not need the function to be 100% to help a Cf'er . This doc could explain this so much better than me right now. Anyway, the drug PCT124 is a particular therapy that works on nonsense mutations such as W1282X that is in clinical trials that is targeted to turn on a pathway to make these sodium/chloride channels work better. I have not heard about this specifically in our clinic we go to now. But it's great they are trying to get data from you. Hopefully this would be treatment that works.
 

Rebjane

Super Moderator
I think this is very interesting. When my daughter was first diagnosed with CF over 4 years ago, a CF doctor said to us that someday in the future this would happen. That the particular mutation/ or mutations you have would be treated specifically and a Cf'er may be on one drug for each particular mutation. For example, my daughter has delta F08 and W1282X. So she could potentially be treated with drugs to target channels that open the sodium chloride channels in each of those particular pathways/mutations. He said you would not need the function to be 100% to help a Cf'er . This doc could explain this so much better than me right now. Anyway, the drug PCT124 is a particular therapy that works on nonsense mutations such as W1282X that is in clinical trials that is targeted to turn on a pathway to make these sodium/chloride channels work better. I have not heard about this specifically in our clinic we go to now. But it's great they are trying to get data from you. Hopefully this would be treatment that works.
 
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tammykrumrey

Guest
Maybe they would taylor it to the known mutation, if there is at least one?
 
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tammykrumrey

Guest
Maybe they would taylor it to the known mutation, if there is at least one?
 
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tammykrumrey

Guest
Maybe they would taylor it to the known mutation, if there is at least one?
 
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Mommafirst

Guest
This is very interesting, Tammy. But it does seem contradictory to the repeated messages we have been getting that genetic mutations aren't a predictor of anything except pancreatic sufficiency. I am quite interested in seeing where this goes. Our doctor recently said to us that my daughter's first year didn't go at all as he expected based on her mutation combination.

Thanks for posting about this. I'd love to hear updates as you hear more.
 
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Mommafirst

Guest
This is very interesting, Tammy. But it does seem contradictory to the repeated messages we have been getting that genetic mutations aren't a predictor of anything except pancreatic sufficiency. I am quite interested in seeing where this goes. Our doctor recently said to us that my daughter's first year didn't go at all as he expected based on her mutation combination.

Thanks for posting about this. I'd love to hear updates as you hear more.
 
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