Breaking News!!!!!!!!!!!!!!!!!!!!!!!

[JennifersHope]

I got this email as well, the thing I wondering is they are letting ppl in the trial with the DF508 gene which I am so thrilled about for everyone that can particpate..

I don't have that gene, do we know if the drug is specifically targeted for that gene, or will it help all CFers? I would think they are using that gene because it is so well known and most common, but I think also that anything that is going to improve the chloride channel will help all Cfers? Does anyone know?

 

[JennifersHope]

I got this email as well, the thing I wondering is they are letting ppl in the trial with the DF508 gene which I am so thrilled about for everyone that can particpate..

I don't have that gene, do we know if the drug is specifically targeted for that gene, or will it help all CFers? I would think they are using that gene because it is so well known and most common, but I think also that anything that is going to improve the chloride channel will help all Cfers? Does anyone know?

 

[JennifersHope]

I got this email as well, the thing I wondering is they are letting ppl in the trial with the DF508 gene which I am so thrilled about for everyone that can particpate..

I don't have that gene, do we know if the drug is specifically targeted for that gene, or will it help all CFers? I would think they are using that gene because it is so well known and most common, but I think also that anything that is going to improve the chloride channel will help all Cfers? Does anyone know?

 

[JennifersHope]

I got this email as well, the thing I wondering is they are letting ppl in the trial with the DF508 gene which I am so thrilled about for everyone that can particpate..

I don't have that gene, do we know if the drug is specifically targeted for that gene, or will it help all CFers? I would think they are using that gene because it is so well known and most common, but I think also that anything that is going to improve the chloride channel will help all Cfers? Does anyone know?

 

[JennifersHope]

I got this email as well, the thing I wondering is they are letting ppl in the trial with the DF508 gene which I am so thrilled about for everyone that can particpate..
<br />
<br />I don't have that gene, do we know if the drug is specifically targeted for that gene, or will it help all CFers? I would think they are using that gene because it is so well known and most common, but I think also that anything that is going to improve the chloride channel will help all Cfers? Does anyone know?

 

[NYCLawGirl]

Actually Jennifer, no. I think this drug specifically targets the DF508 mutation (much like the other Vertex drug specifically targets G551D). This was pulled from a press release and news story re: the drug:

"VX-809 is a novel oral CFTR corrector designed to increase the concentration of F508del CFTR proteins at the cell surface. Vertex completed multiple Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and early 2009." (<a target=_blank class=ftalternatingbarlinklarge href="http://www.foxbusiness.com/story/markets/industries/health-care/vertex-pharmaceuticals-initiates-phase--development-cftr-corrector-vx--patients/)
">http://www.foxbusiness.com/sto...rector-vx--patients/)
</a>
In other words, the drug replinishes the proteins that are absent in CFers with the DF508 mutation. While that doesn't necessarily man it won't help others at all, it won't correct the inherent defect in patients with any other mutations, or at least that's my understanding.

Keep in mind that even patients who are heterozygous for DF508 would be helped by this though, since you only need one functioning CFTR. I know that doesn't help much for people who have no DF508s, but I just want to stress that this drug isn't just for double deltas, although the patient population for the next phase of the study is limited to DDF508 patients.

 

[NYCLawGirl]

Actually Jennifer, no. I think this drug specifically targets the DF508 mutation (much like the other Vertex drug specifically targets G551D). This was pulled from a press release and news story re: the drug:

"VX-809 is a novel oral CFTR corrector designed to increase the concentration of F508del CFTR proteins at the cell surface. Vertex completed multiple Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and early 2009." (<a target=_blank class=ftalternatingbarlinklarge href="http://www.foxbusiness.com/story/markets/industries/health-care/vertex-pharmaceuticals-initiates-phase--development-cftr-corrector-vx--patients/)
">http://www.foxbusiness.com/sto...rector-vx--patients/)
</a>
In other words, the drug replinishes the proteins that are absent in CFers with the DF508 mutation. While that doesn't necessarily man it won't help others at all, it won't correct the inherent defect in patients with any other mutations, or at least that's my understanding.

Keep in mind that even patients who are heterozygous for DF508 would be helped by this though, since you only need one functioning CFTR. I know that doesn't help much for people who have no DF508s, but I just want to stress that this drug isn't just for double deltas, although the patient population for the next phase of the study is limited to DDF508 patients.

 

[NYCLawGirl]

Actually Jennifer, no. I think this drug specifically targets the DF508 mutation (much like the other Vertex drug specifically targets G551D). This was pulled from a press release and news story re: the drug:

"VX-809 is a novel oral CFTR corrector designed to increase the concentration of F508del CFTR proteins at the cell surface. Vertex completed multiple Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and early 2009." (<a target=_blank class=ftalternatingbarlinklarge href="http://www.foxbusiness.com/story/markets/industries/health-care/vertex-pharmaceuticals-initiates-phase--development-cftr-corrector-vx--patients/)
">http://www.foxbusiness.com/sto...rector-vx--patients/)
</a>
In other words, the drug replinishes the proteins that are absent in CFers with the DF508 mutation. While that doesn't necessarily man it won't help others at all, it won't correct the inherent defect in patients with any other mutations, or at least that's my understanding.

Keep in mind that even patients who are heterozygous for DF508 would be helped by this though, since you only need one functioning CFTR. I know that doesn't help much for people who have no DF508s, but I just want to stress that this drug isn't just for double deltas, although the patient population for the next phase of the study is limited to DDF508 patients.

 

[NYCLawGirl]

Actually Jennifer, no. I think this drug specifically targets the DF508 mutation (much like the other Vertex drug specifically targets G551D). This was pulled from a press release and news story re: the drug:

"VX-809 is a novel oral CFTR corrector designed to increase the concentration of F508del CFTR proteins at the cell surface. Vertex completed multiple Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and early 2009." (<a target=_blank class=ftalternatingbarlinklarge href="http://www.foxbusiness.com/story/markets/industries/health-care/vertex-pharmaceuticals-initiates-phase--development-cftr-corrector-vx--patients/)
">http://www.foxbusiness.com/sto...rector-vx--patients/)
</a>
In other words, the drug replinishes the proteins that are absent in CFers with the DF508 mutation. While that doesn't necessarily man it won't help others at all, it won't correct the inherent defect in patients with any other mutations, or at least that's my understanding.

Keep in mind that even patients who are heterozygous for DF508 would be helped by this though, since you only need one functioning CFTR. I know that doesn't help much for people who have no DF508s, but I just want to stress that this drug isn't just for double deltas, although the patient population for the next phase of the study is limited to DDF508 patients.

 

[NYCLawGirl]

Actually Jennifer, no. I think this drug specifically targets the DF508 mutation (much like the other Vertex drug specifically targets G551D). This was pulled from a press release and news story re: the drug:
<br />
<br />"VX-809 is a novel oral CFTR corrector designed to increase the concentration of F508del CFTR proteins at the cell surface. Vertex completed multiple Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and early 2009." (<a target=_blank class=ftalternatingbarlinklarge href="http://www.foxbusiness.com/story/markets/industries/health-care/vertex-pharmaceuticals-initiates-phase--development-cftr-corrector-vx--patients/)
">http://www.foxbusiness.com/sto...rector-vx--patients/)
</a><br />
<br />In other words, the drug replinishes the proteins that are absent in CFers with the DF508 mutation. While that doesn't necessarily man it won't help others at all, it won't correct the inherent defect in patients with any other mutations, or at least that's my understanding.
<br />
<br />Keep in mind that even patients who are heterozygous for DF508 would be helped by this though, since you only need one functioning CFTR. I know that doesn't help much for people who have no DF508s, but I just want to stress that this drug isn't just for double deltas, although the patient population for the next phase of the study is limited to DDF508 patients.

 

[Landy]

This may be a stupid question, but I assume that this drug is a pill?

 

[Landy]

This may be a stupid question, but I assume that this drug is a pill?

 

[Landy]

This may be a stupid question, but I assume that this drug is a pill?

 

[Landy]

This may be a stupid question, but I assume that this drug is a pill?

 

[Landy]

This may be a stupid question, but I assume that this drug is a pill?

 

[Jeana]

It is an oral capsule taken once every 28 days. I read this on saveferris2009's twitter. Click above and scroll down to article. I was disappointed to note that Alaska was not recruiting participants in the study. Of course, if they were, I would have to decide whether to stop breastfeeding and participate. Anyway, I was very excited by many of the articles on saveferris's twitter.

 

[Jeana]

It is an oral capsule taken once every 28 days. I read this on saveferris2009's twitter. Click above and scroll down to article. I was disappointed to note that Alaska was not recruiting participants in the study. Of course, if they were, I would have to decide whether to stop breastfeeding and participate. Anyway, I was very excited by many of the articles on saveferris's twitter.

 

[Jeana]

It is an oral capsule taken once every 28 days. I read this on saveferris2009's twitter. Click above and scroll down to article. I was disappointed to note that Alaska was not recruiting participants in the study. Of course, if they were, I would have to decide whether to stop breastfeeding and participate. Anyway, I was very excited by many of the articles on saveferris's twitter.

 

[Jeana]

It is an oral capsule taken once every 28 days. I read this on saveferris2009's twitter. Click above and scroll down to article. I was disappointed to note that Alaska was not recruiting participants in the study. Of course, if they were, I would have to decide whether to stop breastfeeding and participate. Anyway, I was very excited by many of the articles on saveferris's twitter.

 

[Jeana]

It is an oral capsule taken once every 28 days. I read this on saveferris2009's twitter. Click above and scroll down to article. I was disappointed to note that Alaska was not recruiting participants in the study. Of course, if they were, I would have to decide whether to stop breastfeeding and participate. Anyway, I was very excited by many of the articles on saveferris's twitter.