VX-809 for DDF508

R

ReneeP

New member
I was at the CF Center yesterday for a checkup with my daughters and our CF doc told us that they are going to be starting Phase 2 trials of the VX-809 drug specifically for people with Double Delta F508 this spring. She wants my 14 year old to participate. (the minimum age is 12 so my other daughter can't do it).

I am not going to pretend to understand all of the medical terminology, but to summarize, she said that this is the by far the closest they have come to a cure. She was very excited about it. She said that studies have shown that after taking this medication, people with DDF508 have had completely normal sweat tests, showing that the drug actually corrects the CFTR protein.

This is very similar to the VX-770 which specifically targets the G551D mutation.

Has anyone else heard about this? I am very excited...and hopeful!
 
R

ReneeP

New member
I was at the CF Center yesterday for a checkup with my daughters and our CF doc told us that they are going to be starting Phase 2 trials of the VX-809 drug specifically for people with Double Delta F508 this spring. She wants my 14 year old to participate. (the minimum age is 12 so my other daughter can't do it).

I am not going to pretend to understand all of the medical terminology, but to summarize, she said that this is the by far the closest they have come to a cure. She was very excited about it. She said that studies have shown that after taking this medication, people with DDF508 have had completely normal sweat tests, showing that the drug actually corrects the CFTR protein.

This is very similar to the VX-770 which specifically targets the G551D mutation.

Has anyone else heard about this? I am very excited...and hopeful!
 
R

ReneeP

New member
I was at the CF Center yesterday for a checkup with my daughters and our CF doc told us that they are going to be starting Phase 2 trials of the VX-809 drug specifically for people with Double Delta F508 this spring. She wants my 14 year old to participate. (the minimum age is 12 so my other daughter can't do it).

I am not going to pretend to understand all of the medical terminology, but to summarize, she said that this is the by far the closest they have come to a cure. She was very excited about it. She said that studies have shown that after taking this medication, people with DDF508 have had completely normal sweat tests, showing that the drug actually corrects the CFTR protein.

This is very similar to the VX-770 which specifically targets the G551D mutation.

Has anyone else heard about this? I am very excited...and hopeful!
 
R

ReneeP

New member
I was at the CF Center yesterday for a checkup with my daughters and our CF doc told us that they are going to be starting Phase 2 trials of the VX-809 drug specifically for people with Double Delta F508 this spring. She wants my 14 year old to participate. (the minimum age is 12 so my other daughter can't do it).

I am not going to pretend to understand all of the medical terminology, but to summarize, she said that this is the by far the closest they have come to a cure. She was very excited about it. She said that studies have shown that after taking this medication, people with DDF508 have had completely normal sweat tests, showing that the drug actually corrects the CFTR protein.

This is very similar to the VX-770 which specifically targets the G551D mutation.

Has anyone else heard about this? I am very excited...and hopeful!
 
R

ReneeP

New member
I was at the CF Center yesterday for a checkup with my daughters and our CF doc told us that they are going to be starting Phase 2 trials of the VX-809 drug specifically for people with Double Delta F508 this spring. She wants my 14 year old to participate. (the minimum age is 12 so my other daughter can't do it).
<br />
<br />I am not going to pretend to understand all of the medical terminology, but to summarize, she said that this is the by far the closest they have come to a cure. She was very excited about it. She said that studies have shown that after taking this medication, people with DDF508 have had completely normal sweat tests, showing that the drug actually corrects the CFTR protein.
<br />
<br />This is very similar to the VX-770 which specifically targets the G551D mutation.
<br />
<br />Has anyone else heard about this? I am very excited...and hopeful!
 
H

hmw

New member
I was a bit lost on the terminology too, but we were at a CF conference a couple weeks ago and the keynote speaker was someone from CF national who specializes in the med pipeline... and I do remember her talking about a medication that was normalizing sweat tests. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I am so excited for your dd and hope that you will be posting updates on how she is responding to it!!!

Only double Df508's can participate? Emily has one DF508; we don't know her second mutation. It is hopeful though, even if it is only for DDF508's, since that mutation is such a majority of the CF population. Hopefully it will, if successful, lead to more discoveries that will benefit other mutations. <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
H

hmw

New member
I was a bit lost on the terminology too, but we were at a CF conference a couple weeks ago and the keynote speaker was someone from CF national who specializes in the med pipeline... and I do remember her talking about a medication that was normalizing sweat tests. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I am so excited for your dd and hope that you will be posting updates on how she is responding to it!!!

Only double Df508's can participate? Emily has one DF508; we don't know her second mutation. It is hopeful though, even if it is only for DDF508's, since that mutation is such a majority of the CF population. Hopefully it will, if successful, lead to more discoveries that will benefit other mutations. <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
H

hmw

New member
I was a bit lost on the terminology too, but we were at a CF conference a couple weeks ago and the keynote speaker was someone from CF national who specializes in the med pipeline... and I do remember her talking about a medication that was normalizing sweat tests. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I am so excited for your dd and hope that you will be posting updates on how she is responding to it!!!

Only double Df508's can participate? Emily has one DF508; we don't know her second mutation. It is hopeful though, even if it is only for DDF508's, since that mutation is such a majority of the CF population. Hopefully it will, if successful, lead to more discoveries that will benefit other mutations. <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
H

hmw

New member
I was a bit lost on the terminology too, but we were at a CF conference a couple weeks ago and the keynote speaker was someone from CF national who specializes in the med pipeline... and I do remember her talking about a medication that was normalizing sweat tests. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I am so excited for your dd and hope that you will be posting updates on how she is responding to it!!!

Only double Df508's can participate? Emily has one DF508; we don't know her second mutation. It is hopeful though, even if it is only for DDF508's, since that mutation is such a majority of the CF population. Hopefully it will, if successful, lead to more discoveries that will benefit other mutations. <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
H

hmw

New member
I was a bit lost on the terminology too, but we were at a CF conference a couple weeks ago and the keynote speaker was someone from CF national who specializes in the med pipeline... and I do remember her talking about a medication that was normalizing sweat tests. <img src="i/expressions/face-icon-small-smile.gif" border="0"> I am so excited for your dd and hope that you will be posting updates on how she is responding to it!!!
<br />
<br />Only double Df508's can participate? Emily has one DF508; we don't know her second mutation. It is hopeful though, even if it is only for DDF508's, since that mutation is such a majority of the CF population. Hopefully it will, if successful, lead to more discoveries that will benefit other mutations. <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
R

ReneeP

New member
From what the doctor said to us, the upcoming study is going to be on people with DDF508... however, I found the following article that said that Phase 1 included people with only 1 DF508 mutation... You may want to ask your doctor about it. It's certainly worth checking into!


<a target=_blank class=ftalternatingbarlinklarge href="http://www.reuters.com/finance/stocks/companyProfile?symbol=VRTX.O">LINK</a>VX-809


The Company is evaluating VX-809, an investigational corrector compound designed to increase the concentration of CFTR proteins on the cell surface in patients with CFTR mutations that result in trafficking defects. Vertex has completed two Phase I clinical trials of VX-809 in healthy volunteers. The first clinical trial was a single and multiple-dose trial. The second was a single-dose clinical trial examining the pharmacokinetics and safety of a solid dosage form of VX-809. It has completed an escalating dose pharmacokinetics and safety Phase 1 trial of VX-809 in patients with CF who carry the F508del mutation on at least one of the two alleles.
 
R

ReneeP

New member
From what the doctor said to us, the upcoming study is going to be on people with DDF508... however, I found the following article that said that Phase 1 included people with only 1 DF508 mutation... You may want to ask your doctor about it. It's certainly worth checking into!


<a target=_blank class=ftalternatingbarlinklarge href="http://www.reuters.com/finance/stocks/companyProfile?symbol=VRTX.O">LINK</a>VX-809


The Company is evaluating VX-809, an investigational corrector compound designed to increase the concentration of CFTR proteins on the cell surface in patients with CFTR mutations that result in trafficking defects. Vertex has completed two Phase I clinical trials of VX-809 in healthy volunteers. The first clinical trial was a single and multiple-dose trial. The second was a single-dose clinical trial examining the pharmacokinetics and safety of a solid dosage form of VX-809. It has completed an escalating dose pharmacokinetics and safety Phase 1 trial of VX-809 in patients with CF who carry the F508del mutation on at least one of the two alleles.
 
R

ReneeP

New member
From what the doctor said to us, the upcoming study is going to be on people with DDF508... however, I found the following article that said that Phase 1 included people with only 1 DF508 mutation... You may want to ask your doctor about it. It's certainly worth checking into!


<a target=_blank class=ftalternatingbarlinklarge href="http://www.reuters.com/finance/stocks/companyProfile?symbol=VRTX.O">LINK</a>VX-809


The Company is evaluating VX-809, an investigational corrector compound designed to increase the concentration of CFTR proteins on the cell surface in patients with CFTR mutations that result in trafficking defects. Vertex has completed two Phase I clinical trials of VX-809 in healthy volunteers. The first clinical trial was a single and multiple-dose trial. The second was a single-dose clinical trial examining the pharmacokinetics and safety of a solid dosage form of VX-809. It has completed an escalating dose pharmacokinetics and safety Phase 1 trial of VX-809 in patients with CF who carry the F508del mutation on at least one of the two alleles.
 
R

ReneeP

New member
From what the doctor said to us, the upcoming study is going to be on people with DDF508... however, I found the following article that said that Phase 1 included people with only 1 DF508 mutation... You may want to ask your doctor about it. It's certainly worth checking into!


<a target=_blank class=ftalternatingbarlinklarge href="http://www.reuters.com/finance/stocks/companyProfile?symbol=VRTX.O">LINK</a>VX-809


The Company is evaluating VX-809, an investigational corrector compound designed to increase the concentration of CFTR proteins on the cell surface in patients with CFTR mutations that result in trafficking defects. Vertex has completed two Phase I clinical trials of VX-809 in healthy volunteers. The first clinical trial was a single and multiple-dose trial. The second was a single-dose clinical trial examining the pharmacokinetics and safety of a solid dosage form of VX-809. It has completed an escalating dose pharmacokinetics and safety Phase 1 trial of VX-809 in patients with CF who carry the F508del mutation on at least one of the two alleles.
 
R

ReneeP

New member
From what the doctor said to us, the upcoming study is going to be on people with DDF508... however, I found the following article that said that Phase 1 included people with only 1 DF508 mutation... You may want to ask your doctor about it. It's certainly worth checking into!
<br />
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.reuters.com/finance/stocks/companyProfile?symbol=VRTX.O">LINK</a>VX-809
<br />
<br />
<br />The Company is evaluating VX-809, an investigational corrector compound designed to increase the concentration of CFTR proteins on the cell surface in patients with CFTR mutations that result in trafficking defects. Vertex has completed two Phase I clinical trials of VX-809 in healthy volunteers. The first clinical trial was a single and multiple-dose trial. The second was a single-dose clinical trial examining the pharmacokinetics and safety of a solid dosage form of VX-809. It has completed an escalating dose pharmacokinetics and safety Phase 1 trial of VX-809 in patients with CF who carry the F508del mutation on at least one of the two alleles.
<br />
<br />
 
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