Encouraging Interview/Article about VX-770 and VX-809

dasjsmum

New member
Thanks for posting this. We (my two oldest) are currently on the list for participation in the Vertex770 here in Sydney Australia.My 12yr old has diabetes and 100% FEV1 so wont be able to participate, but I'll still try to get him into it.

The trial here begins recruiting in Dec. Thanks to Loulou and Amy for that info, without their imput I would not have been aware of that. This is incredibly exciting for my family (and yours). I will keep you updated on the progress.

This is the most exciting thing I have heard of in the thirty years we have been involved in cf. I am trying not to get too excited about it though...but I am <img src="i/expressions/face-icon-small-smile.gif" border="0">
 

dasjsmum

New member
Thanks for posting this. We (my two oldest) are currently on the list for participation in the Vertex770 here in Sydney Australia.My 12yr old has diabetes and 100% FEV1 so wont be able to participate, but I'll still try to get him into it.

The trial here begins recruiting in Dec. Thanks to Loulou and Amy for that info, without their imput I would not have been aware of that. This is incredibly exciting for my family (and yours). I will keep you updated on the progress.

This is the most exciting thing I have heard of in the thirty years we have been involved in cf. I am trying not to get too excited about it though...but I am <img src="i/expressions/face-icon-small-smile.gif" border="0">
 

dasjsmum

New member
Thanks for posting this. We (my two oldest) are currently on the list for participation in the Vertex770 here in Sydney Australia.My 12yr old has diabetes and 100% FEV1 so wont be able to participate, but I'll still try to get him into it.

The trial here begins recruiting in Dec. Thanks to Loulou and Amy for that info, without their imput I would not have been aware of that. This is incredibly exciting for my family (and yours). I will keep you updated on the progress.

This is the most exciting thing I have heard of in the thirty years we have been involved in cf. I am trying not to get too excited about it though...but I am <img src="i/expressions/face-icon-small-smile.gif" border="0">
 

dasjsmum

New member
Thanks for posting this. We (my two oldest) are currently on the list for participation in the Vertex770 here in Sydney Australia.My 12yr old has diabetes and 100% FEV1 so wont be able to participate, but I'll still try to get him into it.

The trial here begins recruiting in Dec. Thanks to Loulou and Amy for that info, without their imput I would not have been aware of that. This is incredibly exciting for my family (and yours). I will keep you updated on the progress.

This is the most exciting thing I have heard of in the thirty years we have been involved in cf. I am trying not to get too excited about it though...but I am <img src="i/expressions/face-icon-small-smile.gif" border="0">
 

dasjsmum

New member
Thanks for posting this. We (my two oldest) are currently on the list for participation in the Vertex770 here in Sydney Australia.My 12yr old has diabetes and 100% FEV1 so wont be able to participate, but I'll still try to get him into it.
<br />
<br />The trial here begins recruiting in Dec. Thanks to Loulou and Amy for that info, without their imput I would not have been aware of that. This is incredibly exciting for my family (and yours). I will keep you updated on the progress.
<br />
<br />This is the most exciting thing I have heard of in the thirty years we have been involved in cf. I am trying not to get too excited about it though...but I am <img src="i/expressions/face-icon-small-smile.gif" border="0">
 

Jeana

New member
I thought I'd post this again for those that may not have seen it previously. Hope this helps!

They are doing a clinical trial to see if VX-770 works on Delta F508, as well. Our clinic is participating.

<b>According to the CFF website</b>:

"VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.

Why wasn't the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?

Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770."

This means, that if the CFTR was located at the cell surface, it might work for Delta F508. So, if we use the VX-809 and the VX-770, Delta F508s might have very good results, since...

"Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."

*In other words, VX-809 should move the CFTR to the cell surface and VX-770 should correct the CFTR at the cell surface.*

<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/">http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/</a>
 

Jeana

New member
I thought I'd post this again for those that may not have seen it previously. Hope this helps!

They are doing a clinical trial to see if VX-770 works on Delta F508, as well. Our clinic is participating.

<b>According to the CFF website</b>:

"VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.

Why wasn't the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?

Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770."

This means, that if the CFTR was located at the cell surface, it might work for Delta F508. So, if we use the VX-809 and the VX-770, Delta F508s might have very good results, since...

"Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."

*In other words, VX-809 should move the CFTR to the cell surface and VX-770 should correct the CFTR at the cell surface.*

<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/">http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/</a>
 

Jeana

New member
I thought I'd post this again for those that may not have seen it previously. Hope this helps!

They are doing a clinical trial to see if VX-770 works on Delta F508, as well. Our clinic is participating.

<b>According to the CFF website</b>:

"VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.

Why wasn't the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?

Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770."

This means, that if the CFTR was located at the cell surface, it might work for Delta F508. So, if we use the VX-809 and the VX-770, Delta F508s might have very good results, since...

"Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."

*In other words, VX-809 should move the CFTR to the cell surface and VX-770 should correct the CFTR at the cell surface.*

<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/">http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/</a>
 

Jeana

New member
I thought I'd post this again for those that may not have seen it previously. Hope this helps!

They are doing a clinical trial to see if VX-770 works on Delta F508, as well. Our clinic is participating.

<b>According to the CFF website</b>:

"VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.

Why wasn't the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?

Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770."

This means, that if the CFTR was located at the cell surface, it might work for Delta F508. So, if we use the VX-809 and the VX-770, Delta F508s might have very good results, since...

"Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."

*In other words, VX-809 should move the CFTR to the cell surface and VX-770 should correct the CFTR at the cell surface.*

<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/">http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/</a>
 

Jeana

New member
I thought I'd post this again for those that may not have seen it previously. Hope this helps!
<br />
<br />They are doing a clinical trial to see if VX-770 works on Delta F508, as well. Our clinic is participating.
<br />
<br /><b>According to the CFF website</b>:
<br />
<br />"VX-770, the most advanced of the two compounds, is known as a potentiator and is designed to allow CFTR located at the cell surface to function correctly.
<br />
<br />Why wasn't the Delta F508 mutation chosen for the Phase 2 VX-770 clinical trials?
<br />
<br />Because G551D is already located at the cell surface, these patients have the greatest potential to benefit from treatment with VX-770."
<br />
<br />This means, that if the CFTR was located at the cell surface, it might work for Delta F508. So, if we use the VX-809 and the VX-770, Delta F508s might have very good results, since...
<br />
<br />"Vertex is also developing VX-809, known as a corrector, which is designed to move defective CFTR to its proper place in the cell."
<br />
<br />*In other words, VX-809 should move the CFTR to the cell surface and VX-770 should correct the CFTR at the cell surface.*
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/">http://www.cff.org/research/Cl...lResearch/FAQs/VX-770/</a>
<br />
<br />
 
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