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A "cure"? Please explain...

K

kitomd21

New member
A

Here's one that mentions being mutation-specific (also from CFF website)...

VX-770 (Vertex Pharmaceuticals) - VX-770 is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
 
K

kitomd21

New member
A

Here's one that mentions being mutation-specific (also from CFF website)...

VX-770 (Vertex Pharmaceuticals) - VX-770 is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
 
K

kitomd21

New member
A

Here's one that mentions being mutation-specific (also from CFF website)...

VX-770 (Vertex Pharmaceuticals) - VX-770 is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
 
K

kitomd21

New member
A

Here's one that mentions being mutation-specific (also from CFF website)...

VX-770 (Vertex Pharmaceuticals) - VX-770 is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
 
K

kitomd21

New member
A

Here's one that mentions being mutation-specific (also from CFF website)...
<br />
<br />VX-770 (Vertex Pharmaceuticals) - VX-770 is a new compound called a "potentiator" that may act upon the CFTR protein and help to open the chloride channel in CF cells. Phase 1 dosing has been completed in patients. Researchers are evaluating results and a Phase 2 trial has begun in CF patients.
<br />
<br />The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
 
R

Ratatosk

Administrator
Staff member
A

Somewhere there's a post about the VX-770 and I think I posted a link about another version for those with the Double delta f508 gene. It's in a phase 1 study. Now THAT would be exciting news for us!

<a target=_blank class=ftalternatingbarlinklarge href="http://www.vrtx.com/current-projects/drug-candidates/VX-809.html">http://www.vrtx.com/current-pr...candidates/VX-809.html</a>
 
R

Ratatosk

Administrator
Staff member
A

Somewhere there's a post about the VX-770 and I think I posted a link about another version for those with the Double delta f508 gene. It's in a phase 1 study. Now THAT would be exciting news for us!

<a target=_blank class=ftalternatingbarlinklarge href="http://www.vrtx.com/current-projects/drug-candidates/VX-809.html">http://www.vrtx.com/current-pr...candidates/VX-809.html</a>
 
R

Ratatosk

Administrator
Staff member
A

Somewhere there's a post about the VX-770 and I think I posted a link about another version for those with the Double delta f508 gene. It's in a phase 1 study. Now THAT would be exciting news for us!

<a target=_blank class=ftalternatingbarlinklarge href="http://www.vrtx.com/current-projects/drug-candidates/VX-809.html">http://www.vrtx.com/current-pr...candidates/VX-809.html</a>
 
R

Ratatosk

Administrator
Staff member
A

Somewhere there's a post about the VX-770 and I think I posted a link about another version for those with the Double delta f508 gene. It's in a phase 1 study. Now THAT would be exciting news for us!

<a target=_blank class=ftalternatingbarlinklarge href="http://www.vrtx.com/current-projects/drug-candidates/VX-809.html">http://www.vrtx.com/current-pr...candidates/VX-809.html</a>
 
R

Ratatosk

Administrator
Staff member
A

Somewhere there's a post about the VX-770 and I think I posted a link about another version for those with the Double delta f508 gene. It's in a phase 1 study. Now THAT would be exciting news for us!
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.vrtx.com/current-projects/drug-candidates/VX-809.html">http://www.vrtx.com/current-pr...candidates/VX-809.html</a>
 
T

tammykrumrey

Guest
A

The kids CF doc said it is not a cure, but a maintaince drug that would be taken daily. And the drug that is in the pipeline to help the delF508 is great for so many people! If you have only one delF508 and another mutation, it will work for you. That helps the majority of those with CF.
The girls doc said that he could possibly be asking Kayla and Hannah to be participating in studies as early as next year for this (they have delF508 and 1898+1G>A).
 
T

tammykrumrey

Guest
A

The kids CF doc said it is not a cure, but a maintaince drug that would be taken daily. And the drug that is in the pipeline to help the delF508 is great for so many people! If you have only one delF508 and another mutation, it will work for you. That helps the majority of those with CF.
The girls doc said that he could possibly be asking Kayla and Hannah to be participating in studies as early as next year for this (they have delF508 and 1898+1G>A).
 
T

tammykrumrey

Guest
A

The kids CF doc said it is not a cure, but a maintaince drug that would be taken daily. And the drug that is in the pipeline to help the delF508 is great for so many people! If you have only one delF508 and another mutation, it will work for you. That helps the majority of those with CF.
The girls doc said that he could possibly be asking Kayla and Hannah to be participating in studies as early as next year for this (they have delF508 and 1898+1G>A).
 
T

tammykrumrey

Guest
A

The kids CF doc said it is not a cure, but a maintaince drug that would be taken daily. And the drug that is in the pipeline to help the delF508 is great for so many people! If you have only one delF508 and another mutation, it will work for you. That helps the majority of those with CF.
The girls doc said that he could possibly be asking Kayla and Hannah to be participating in studies as early as next year for this (they have delF508 and 1898+1G>A).
 
T

tammykrumrey

Guest
A

The kids CF doc said it is not a cure, but a maintaince drug that would be taken daily. And the drug that is in the pipeline to help the delF508 is great for so many people! If you have only one delF508 and another mutation, it will work for you. That helps the majority of those with CF.
<br />The girls doc said that he could possibly be asking Kayla and Hannah to be participating in studies as early as next year for this (they have delF508 and 1898+1G>A).
 
J

jdprecious

New member
A

Jae is in the Denufosol study. It is absolutly NOT a cure for CF, however makes it more manageable by allowing the lungs to function properly in the production of salt on the CFTR .
 
J

jdprecious

New member
A

Jae is in the Denufosol study. It is absolutly NOT a cure for CF, however makes it more manageable by allowing the lungs to function properly in the production of salt on the CFTR .
 
J

jdprecious

New member
A

Jae is in the Denufosol study. It is absolutly NOT a cure for CF, however makes it more manageable by allowing the lungs to function properly in the production of salt on the CFTR .
 
J

jdprecious

New member
A

Jae is in the Denufosol study. It is absolutly NOT a cure for CF, however makes it more manageable by allowing the lungs to function properly in the production of salt on the CFTR .
 
J

jdprecious

New member
A

Jae is in the Denufosol study. It is absolutly NOT a cure for CF, however makes it more manageable by allowing the lungs to function properly in the production of salt on the CFTR .
 
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