To those on kalydeco---Lauren (your thoughts, please)

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saveferris2009

New member
<div class="FTQUOTE"><begin quote><em>Originally posted by: <strong>dasjsmum</strong></em> Amy, I thought I read that the 770 might work for your mutation? Is yours one of the gating mutations? If so, have you attempted to get Kalydeco yet? I just thought I read on the FB site I mentioned above that Kalydeco had been approved in the US for other gating mutations (which I thought yours might be).</end quote>

Nope, I have 2 class II's.
Won't stop me though... I'm still going to try it.
I'm so sorry to hear about your daughter in Australia...... I cannot imagine how frustrating that would be to the government say no to a medication.
Take care and have hope! Hugs!
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><em>Originally posted by: <strong>dasjsmum</strong></em> Amy, I thought I read that the 770 might work for your mutation? Is yours one of the gating mutations? If so, have you attempted to get Kalydeco yet? I just thought I read on the FB site I mentioned above that Kalydeco had been approved in the US for other gating mutations (which I thought yours might be).</end quote>

Nope, I have 2 class II's.
Won't stop me though... I'm still going to try it.
I'm so sorry to hear about your daughter in Australia...... I cannot imagine how frustrating that would be to the government say no to a medication.
Take care and have hope! Hugs!
 
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fhayden

New member
Well, I've got CFRD. My local endo insists I have type 1 so I use an insulin pump. I'm going to start taking Kalydeco soon. I've already received my shipment of Kalydeco in the mail but haven't started taking it yet.. Because I've already receive a double lung transplant because of CF I have to coordinate my transplant labs with taking the medication. There are no studies available for people like myself. I'll report back if there is any improvement with my CFRD while taking Kalydeco. My CF doc doubts Kalydeco will help with CFRD. Frank Hayden
 
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fhayden

New member
Well, I've got CFRD. My local endo insists I have type 1 so I use an insulin pump. I'm going to start taking Kalydeco soon. I've already received my shipment of Kalydeco in the mail but haven't started taking it yet.. Because I've already receive a double lung transplant because of CF I have to coordinate my transplant labs with taking the medication. There are no studies available for people like myself. I'll report back if there is any improvement with my CFRD while taking Kalydeco. My CF doc doubts Kalydeco will help with CFRD. Frank Hayden
 
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dasjsmum

New member
Frank, why do you think you have cfrd when your endo says you have Type 1? There is a test for Type 1. Even given what Amy says above (which is pretty interesting, thanks Amy), Type 1 is obvious because it's due to an auto immune disease that destroys your beta cells and people become insulin dependetn straight away basically.


CFRD insulin needs usually taper and you arent completely insulin dependent to start with.

Your blood will confirm Type 1 if you have it. My son has Type 1 and uses an insulin pump. I have to tell you, the insulin pump is fantastic and he loves it. The benefit for you is that it will help keep your BGLs on a much more even keel which should assist you in keeping infection under control. I personlly still think Kalydeco might help with cfrd, but dont know, it will be interesting to see <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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dasjsmum

New member
Frank, why do you think you have cfrd when your endo says you have Type 1? There is a test for Type 1. Even given what Amy says above (which is pretty interesting, thanks Amy), Type 1 is obvious because it's due to an auto immune disease that destroys your beta cells and people become insulin dependetn straight away basically.


CFRD insulin needs usually taper and you arent completely insulin dependent to start with.

Your blood will confirm Type 1 if you have it. My son has Type 1 and uses an insulin pump. I have to tell you, the insulin pump is fantastic and he loves it. The benefit for you is that it will help keep your BGLs on a much more even keel which should assist you in keeping infection under control. I personlly still think Kalydeco might help with cfrd, but dont know, it will be interesting to see <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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fhayden

New member
First, I'm 42 yrs old and wasn't diagnosed unil 2005. I was diagnosed with blood tests at UPMC and was told I have CFRD. My pancrease still makes some insulin and my sensitivies flucutate. I chose to use the pump because I got too many lows using lantus or pills.
There is a doctor who is the guru of CFRD and his name is Dr. Stalvey. He used to work at U of Florida but now works at a hospital in Maryland. Dr. Stalvey specializes in CFRD. He was my doctor for a short while before moving away. He even told me I have CFRD through blood tests. I don't know why my current endo thinks all CFRD are type 1 but she does. I've actually been to serveral endos in my area and still have issues conving them CFRD in not just a type 1 but its own type of diabetes.
Frank Hayden
 
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fhayden

New member
First, I'm 42 yrs old and wasn't diagnosed unil 2005. I was diagnosed with blood tests at UPMC and was told I have CFRD. My pancrease still makes some insulin and my sensitivies flucutate. I chose to use the pump because I got too many lows using lantus or pills.
There is a doctor who is the guru of CFRD and his name is Dr. Stalvey. He used to work at U of Florida but now works at a hospital in Maryland. Dr. Stalvey specializes in CFRD. He was my doctor for a short while before moving away. He even told me I have CFRD through blood tests. I don't know why my current endo thinks all CFRD are type 1 but she does. I've actually been to serveral endos in my area and still have issues conving them CFRD in not just a type 1 but its own type of diabetes.
Frank Hayden
 
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Incomudrox

New member
I wonder, for those who do not have their bacteria under control but are not full exacerbation stage at the time of start Kalydeco should they be admitted to get a jump on the bacteria? Giving the Kalydeco an edge and a better chance at gaining back lung function quickly?
 
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Incomudrox

New member
I wonder, for those who do not have their bacteria under control but are not full exacerbation stage at the time of start Kalydeco should they be admitted to get a jump on the bacteria? Giving the Kalydeco an edge and a better chance at gaining back lung function quickly?
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>dasjsmum</b></i> Amy, I thought I read that the 770 might work for your mutation? Is yours one of the gating mutations? If so, have you attempted to get Kalydeco yet? I just thought I read on the FB site I mentioned above that Kalydeco had been approved in the US for other gating mutations (which I thought yours might be).</end quote> Nope, I have 2 class II's. Won't stop me though... I'm still going to try it. I'm so sorry to hear about your daughter in Australia...... I cannot imagine how frustrating that would be to the government say no to a medication. Take care and have hope! Hugs!</end quote>

Just to further clarify (and I'm sorry Ms. Mandy to hijack your thread), here is my rationale:

According to Vertex: "<span>In recombinant cells VX-770 increased CFTR channel open probability (P(o)) in both the F508del processing mutation<span>and the G551D gating mutation."
<a href="http://www.ncbi.nlm.nih.gov/pubmed/19846789">http://www.ncbi.nlm.nih.gov/pubmed/19846789</a>


From Annals of Human Genetics, 2003, by Rowntree et al,:
"Measurements of Cl- conductase of intestine and respiratory tissues of DF508 homozygote CF patients suggest, in vivo, that at least some DF508 CFTR can reach the plasma membrane"
<a href="http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf">http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf</a>


Most likely I won't notice any difference, but of course I want to just give it a try
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> <div class="FTQUOTE"><begin quote><i>Originally posted by: <b>dasjsmum</b></i> Amy, I thought I read that the 770 might work for your mutation? Is yours one of the gating mutations? If so, have you attempted to get Kalydeco yet? I just thought I read on the FB site I mentioned above that Kalydeco had been approved in the US for other gating mutations (which I thought yours might be).</end quote> Nope, I have 2 class II's. Won't stop me though... I'm still going to try it. I'm so sorry to hear about your daughter in Australia...... I cannot imagine how frustrating that would be to the government say no to a medication. Take care and have hope! Hugs!</end quote>

Just to further clarify (and I'm sorry Ms. Mandy to hijack your thread), here is my rationale:

According to Vertex: "<span>In recombinant cells VX-770 increased CFTR channel open probability (P(o)) in both the F508del processing mutation<span>and the G551D gating mutation."
<a href="http://www.ncbi.nlm.nih.gov/pubmed/19846789">http://www.ncbi.nlm.nih.gov/pubmed/19846789</a>


From Annals of Human Genetics, 2003, by Rowntree et al,:
"Measurements of Cl- conductase of intestine and respiratory tissues of DF508 homozygote CF patients suggest, in vivo, that at least some DF508 CFTR can reach the plasma membrane"
<a href="http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf">http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf</a>


Most likely I won't notice any difference, but of course I want to just give it a try
 
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jbrandonAW

New member
I started taking the meds one week ago and I have had amazing results in a week. I feel horrible though, but a different kind of horrible. 2 weeks ago I had just finished 2 weeks of bactrium adm cipro and 1 week of levequin and I called my dr and asked for antibiotics and thought I might to be in the hospital. Now I don't feel like I have a lung infection at all. I feel different though, partly good and parrtly bad -or maybe its unknown bc I don't really know what feeling good is like. tHE GREATEST part is the rise in my PFT's in one week. I have an at home FEV1 meter... well I hate to do this but to check out the whole story www.insidecf.blogspot.com .... My blog has my updates on it... amazing truely amazing!
 
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jbrandonAW

New member
I started taking the meds one week ago and I have had amazing results in a week. I feel horrible though, but a different kind of horrible. 2 weeks ago I had just finished 2 weeks of bactrium adm cipro and 1 week of levequin and I called my dr and asked for antibiotics and thought I might to be in the hospital. Now I don't feel like I have a lung infection at all. I feel different though, partly good and parrtly bad -or maybe its unknown bc I don't really know what feeling good is like. tHE GREATEST part is the rise in my PFT's in one week. I have an at home FEV1 meter... well I hate to do this but to check out the whole story www.insidecf.blogspot.com .... My blog has my updates on it... amazing truely amazing!
 
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dasjsmum

New member
Interesting info re the cfrd and Type 1 thanks <img src="i/expressions/face-icon-small-smile.gif" border="0"> Bummer with the class 11, I thought you were a gating mutation. It's coming though <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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dasjsmum

New member
Interesting info re the cfrd and Type 1 thanks <img src="i/expressions/face-icon-small-smile.gif" border="0"> Bummer with the class 11, I thought you were a gating mutation. It's coming though <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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saveferris2009

New member
Ya I'm trying Kalydeco this weekend anyway, though. Probably won't do anything, but it's possible it will have a little impact.

I'll keep you all posted
 
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saveferris2009

New member
Ya I'm trying Kalydeco this weekend anyway, though. Probably won't do anything, but it's possible it will have a little impact.

I'll keep you all posted
 
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