New in the club... :-(

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Kaethe108

Guest
Hi Aleksandra!
<br />Nice to read a "neighbour", although it is still 1300 km between our homes, as we live right next to the French border (exactly 50 metres...) in west Germany.
<br />I now ordered Aquadeks directly from the producer, check out http://www.yasooglobal.com/aquadeks/.
<br />They ship from Cyprus and one bottle (60ml) costs 45 USD.
<br />How much do you pay in Poland?
 
A

AleksandraKaczynska

New member
Hi - it's the same price more or less - exactly 44,5 $ US
But the foundation gets the prices lower if they can - I'll let you know <img src="i/expressions/face-icon-small-smile.gif" border="0"> - one it was a big campain with sponsors and the price was then very low - about 15$ per bottle - but still it's good to know where to buy if they run out. Sometimes we can't buy it an then I turn to less effective vitamins.
Wish we had your health system <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
A

AleksandraKaczynska

New member
Hi - it's the same price more or less - exactly 44,5 $ US
But the foundation gets the prices lower if they can - I'll let you know <img src="i/expressions/face-icon-small-smile.gif" border="0"> - one it was a big campain with sponsors and the price was then very low - about 15$ per bottle - but still it's good to know where to buy if they run out. Sometimes we can't buy it an then I turn to less effective vitamins.
Wish we had your health system <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
A

AleksandraKaczynska

New member
Hi - it's the same price more or less - exactly 44,5 $ US
<br />But the foundation gets the prices lower if they can - I'll let you know <img src="i/expressions/face-icon-small-smile.gif" border="0"> - one it was a big campain with sponsors and the price was then very low - about 15$ per bottle - but still it's good to know where to buy if they run out. Sometimes we can't buy it an then I turn to less effective vitamins.
<br />Wish we had your health system <img src="i/expressions/face-icon-small-smile.gif" border="0">
<br />
 
K

Kaethe108

Guest
Well, in German it is not covered by health insurance, as it has no approval (yet) on the German market....
<br>But as everything else is covered we can live with that extra cost.
<br>
<br>Yes, the system here seems to work well. There is a strong network of experts working together to help with CF patients. We feel in very good hands!
<br>Today we had the first inhaling therapy with the physiotherapist and Livi seemed to like it. She fell asleep while inhaling.
<br>
<br>Ah, and I had a very nice email contact with someone from PTC Therapeutics! They are hoping to bring Ataluren on the market in 2013! :)))<br>That would change also the live of your little daughter! <br>
 
K

Kaethe108

Guest
Well, in German it is not covered by health insurance, as it has no approval (yet) on the German market....
<br>But as everything else is covered we can live with that extra cost.
<br>
<br>Yes, the system here seems to work well. There is a strong network of experts working together to help with CF patients. We feel in very good hands!
<br>Today we had the first inhaling therapy with the physiotherapist and Livi seemed to like it. She fell asleep while inhaling.
<br>
<br>Ah, and I had a very nice email contact with someone from PTC Therapeutics! They are hoping to bring Ataluren on the market in 2013! :)))<br>That would change also the live of your little daughter! <br>
 
K

Kaethe108

Guest
Well, in German it is not covered by health insurance, as it has no approval (yet) on the German market....
<br>But as everything else is covered we can live with that extra cost.
<br>
<br>Yes, the system here seems to work well. There is a strong network of experts working together to help with CF patients. We feel in very good hands!
<br>Today we had the first inhaling therapy with the physiotherapist and Livi seemed to like it. She fell asleep while inhaling.
<br>
<br>Ah, and I had a very nice email contact with someone from PTC Therapeutics! They are hoping to bring Ataluren on the market in 2013! :)))<br>That would change also the live of your little daughter! <br>
 
N

natybariloche

New member
<div>Hi Kaethe. My name is natalia i am from argentina a have a little boy Dante, hi is 9 months and he has the sames moutasions than your kid. Df508/r553x.</div><div>I am visiting here in my country a genetist. Omar Pivetta. If you see google you will find lots of publications. He told me yesterday that there are two drugs in study. One for Df508 vertex 770 and the othr Ataluren. Any of those can be used in ours sons. If you can controll the dissease of one mutation you can be safe.</div><div>My son, as you tell is reciving Aquadeks, you can fins it here, in fipan.Is from Canada. I can buy it for you and send it.</div><div>My son as yours, has little problems of respiration. He has mostly from pancreas. I think maybe is the combination of the moutations. I will ask.</div><div>Send kisses.</div><div>Still in touch, and pardon for my inglish.</div><div>Natalia</div>
 
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natybariloche

New member
Hi Kaethe. My name is natalia i am from argentina a have a little boy Dante, hi is 9 months and he has the sames moutasions than your kid. Df508/r553x.I am visiting here in my country a genetist. Omar Pivetta. If you see google you will find lots of publications. He told me yesterday that there are two drugs in study. One for Df508 vertex 770 and the othr Ataluren. Any of those can be used in ours sons. If you can controll the dissease of one mutation you can be safe.My son, as you tell is reciving Aquadeks, you can fins it here, in fipan.Is from Canada. I can buy it for you and send it.My son as yours, has little problems of respiration. He has mostly from pancreas. I think maybe is the combination of the moutations. I will ask.Send kisses.Still in touch, and pardon for my inglish.Natalia
 
N

natybariloche

New member
Hi Kaethe. My name is natalia i am from argentina a have a little boy Dante, hi is 9 months and he has the sames moutasions than your kid. Df508/r553x.I am visiting here in my country a genetist. Omar Pivetta. If you see google you will find lots of publications. He told me yesterday that there are two drugs in study. One for Df508 vertex 770 and the othr Ataluren. Any of those can be used in ours sons. If you can controll the dissease of one mutation you can be safe.My son, as you tell is reciving Aquadeks, you can fins it here, in fipan.Is from Canada. I can buy it for you and send it.My son as yours, has little problems of respiration. He has mostly from pancreas. I think maybe is the combination of the moutations. I will ask.Send kisses.Still in touch, and pardon for my inglish.Natalia
 
A

AleksandraKaczynska

New member
AquADEKs is not covered here by insurence also - acctually almost nothing is.. even saline...but it was cheeper cause the foundation got some sponsors to cover part of the costs... that's all.
Anyway - if we have it cheepr again I can buy it for you - now it's regular price - only that I get shipment for free from the foundation.
Wish we can make it in good shape till 2013 <img src="i/expressions/face-icon-small-smile.gif" border="0">
Although I'm not sure if Ataluren or Vertex will work for Asia ...
her leadin muation is class 5 - I think I read it will work for her also - but I'm not sure..
If it takes longer here - We'll just have go to Germany while my mom still has friend there...
 
A

AleksandraKaczynska

New member
AquADEKs is not covered here by insurence also - acctually almost nothing is.. even saline...but it was cheeper cause the foundation got some sponsors to cover part of the costs... that's all.
Anyway - if we have it cheepr again I can buy it for you - now it's regular price - only that I get shipment for free from the foundation.
Wish we can make it in good shape till 2013 <img src="i/expressions/face-icon-small-smile.gif" border="0">
Although I'm not sure if Ataluren or Vertex will work for Asia ...
her leadin muation is class 5 - I think I read it will work for her also - but I'm not sure..
If it takes longer here - We'll just have go to Germany while my mom still has friend there...
 
A

AleksandraKaczynska

New member
AquADEKs is not covered here by insurence also - acctually almost nothing is.. even saline...but it was cheeper cause the foundation got some sponsors to cover part of the costs... that's all.
<br />Anyway - if we have it cheepr again I can buy it for you - now it's regular price - only that I get shipment for free from the foundation.
<br />Wish we can make it in good shape till 2013 <img src="i/expressions/face-icon-small-smile.gif" border="0">
<br />Although I'm not sure if Ataluren or Vertex will work for Asia ...
<br />her leadin muation is class 5 - I think I read it will work for her also - but I'm not sure..
<br />If it takes longer here - We'll just have go to Germany while my mom still has friend there...
<br />
<br />
<br />
 
K

Kaethe108

Guest
Dear Natalia,
thanks for your post! "Good" to read somebody with the same mutations, although I am aware that even the same mutations can make different problems.
But from what I've read so far, although both belong to the more severe classes, it looks like F508del/R553X makes more problems with the pancreas and less with the lungs. It is like this also with our daughter. With six weeks she had already elastase <50 and still we are struggling to find the right dosis with enzymes. But her lungs are free. *keep fingers crossed*
Concerning the new drugs, the big hope for our children is Ataluren. Vertex VX-770 targets people with another mutation. But there will be further trials with VX-770 and VX-809, which would help with F508del mutation. I don't know when this would be ready for the market, as they are only in phase 2. see: http://www.vrtx.com/current-projects/drug-candidates/vx-770.html
But as I wrote already in my last post, Ataluren could be on the market already in 2013!
First for children >6 years, but after more trials it will also be available for younger children! Ataluren would help with the R553X mutation!

Dear Aleksandra,
Ataluren would also help Asia, because she has the R553X mutation. The drug is designed to help with all nonsense mutations!
You can read the Q&A on the website: http://www.ptcbio.com/6.1.2_ataluren_CF_trial_FAQ.aspx#Q2
There it says that the drug will help also if somebody has only one nonsense muation. As I understood it is then comparable to a carrier, who has one mutation, but no disease.

The question that is always in my mind is how big the impact of the drug will be...
Will the CF symptoms only get milder or will they disappear completely? I know that probably nobody knows the answer yet, but I allow myself dreming, that these new drugs will "heal" our babies!
 
K

Kaethe108

Guest
Dear Natalia,
thanks for your post! "Good" to read somebody with the same mutations, although I am aware that even the same mutations can make different problems.
But from what I've read so far, although both belong to the more severe classes, it looks like F508del/R553X makes more problems with the pancreas and less with the lungs. It is like this also with our daughter. With six weeks she had already elastase <50 and still we are struggling to find the right dosis with enzymes. But her lungs are free. *keep fingers crossed*
Concerning the new drugs, the big hope for our children is Ataluren. Vertex VX-770 targets people with another mutation. But there will be further trials with VX-770 and VX-809, which would help with F508del mutation. I don't know when this would be ready for the market, as they are only in phase 2. see: http://www.vrtx.com/current-projects/drug-candidates/vx-770.html
But as I wrote already in my last post, Ataluren could be on the market already in 2013!
First for children >6 years, but after more trials it will also be available for younger children! Ataluren would help with the R553X mutation!

Dear Aleksandra,
Ataluren would also help Asia, because she has the R553X mutation. The drug is designed to help with all nonsense mutations!
You can read the Q&A on the website: http://www.ptcbio.com/6.1.2_ataluren_CF_trial_FAQ.aspx#Q2
There it says that the drug will help also if somebody has only one nonsense muation. As I understood it is then comparable to a carrier, who has one mutation, but no disease.

The question that is always in my mind is how big the impact of the drug will be...
Will the CF symptoms only get milder or will they disappear completely? I know that probably nobody knows the answer yet, but I allow myself dreming, that these new drugs will "heal" our babies!
 
K

Kaethe108

Guest
Dear Natalia,
<br />thanks for your post! "Good" to read somebody with the same mutations, although I am aware that even the same mutations can make different problems.
<br />But from what I've read so far, although both belong to the more severe classes, it looks like F508del/R553X makes more problems with the pancreas and less with the lungs. It is like this also with our daughter. With six weeks she had already elastase <50 and still we are struggling to find the right dosis with enzymes. But her lungs are free. *keep fingers crossed*
<br />Concerning the new drugs, the big hope for our children is Ataluren. Vertex VX-770 targets people with another mutation. But there will be further trials with VX-770 and VX-809, which would help with F508del mutation. I don't know when this would be ready for the market, as they are only in phase 2. see: http://www.vrtx.com/current-projects/drug-candidates/vx-770.html
<br />But as I wrote already in my last post, Ataluren could be on the market already in 2013!
<br />First for children >6 years, but after more trials it will also be available for younger children! Ataluren would help with the R553X mutation!
<br />
<br />Dear Aleksandra,
<br />Ataluren would also help Asia, because she has the R553X mutation. The drug is designed to help with all nonsense mutations!
<br />You can read the Q&A on the website: http://www.ptcbio.com/6.1.2_ataluren_CF_trial_FAQ.aspx#Q2
<br />There it says that the drug will help also if somebody has only one nonsense muation. As I understood it is then comparable to a carrier, who has one mutation, but no disease.
<br />
<br />The question that is always in my mind is how big the impact of the drug will be...
<br />Will the CF symptoms only get milder or will they disappear completely? I know that probably nobody knows the answer yet, but I allow myself dreming, that these new drugs will "heal" our babies!
<br />
<br />
<br />
 
T

TerriC

Guest
<br>I just want to tell you that I understand everything that you are going through now with a newly diagnosed baby as I went through that 23 years ago with my daughter.  Just so you know, she too had no lung involvement when she was born and to this day still has very little lung invovlement.  She played rep. soccer and has run full marathons.  I hope this helps you to look to the future with hope and know that when your daughter is 23 there will be even more treatments if not a cure for this disease.   TerriMom of Lindsay 25 w/cf, and Courtney 23 w/cf
 
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