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Just curious
- Thread starter momtohazel
- Start date
K
Kaethe108
Guest
I talked to a doctor who is involved in the Ataluren study in one CF center in Germany.
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
K
Kaethe108
Guest
I talked to a doctor who is involved in the Ataluren study in one CF center in Germany.
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
K
Kaethe108
Guest
I talked to a doctor who is involved in the Ataluren study in one CF center in Germany.
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
He told me that Phase III is finished and it will take about 6 moths to get the results. After this, if the results are positive, it will still take about 1.5 years to get in on the (German) market.
For children younger then 6 years there will have to be an additional trial and my daughter will have the chance to get on the trial...
momtohazel
New member
thanks for the responses everyone...hope to hear even more. this was kinda my point and original question with this topic? why don't we know more?<br><br>our daughter's CF doc hasn't been able to give me any info. when i ask, the doc says, "oh, i'll try to find something out." but when we walk in the door, the first thing the CF nurse says is, "Have you heard the latest about vertex?"<br><br>i think i might contact some of the researchers/clinical sites who participated in conducting the trials and see what info they may have. I did this about a year ago and they were helpful in answering some questions<br>
momtohazel
New member
thanks for the responses everyone...hope to hear even more. this was kinda my point and original question with this topic? why don't we know more?<br><br>our daughter's CF doc hasn't been able to give me any info. when i ask, the doc says, "oh, i'll try to find something out." but when we walk in the door, the first thing the CF nurse says is, "Have you heard the latest about vertex?"<br><br>i think i might contact some of the researchers/clinical sites who participated in conducting the trials and see what info they may have. I did this about a year ago and they were helpful in answering some questions<br>
momtohazel
New member
thanks for the responses everyone...hope to hear even more. this was kinda my point and original question with this topic? why don't we know more?<br><br>our daughter's CF doc hasn't been able to give me any info. when i ask, the doc says, "oh, i'll try to find something out." but when we walk in the door, the first thing the CF nurse says is, "Have you heard the latest about vertex?"<br><br>i think i might contact some of the researchers/clinical sites who participated in conducting the trials and see what info they may have. I did this about a year ago and they were helpful in answering some questions<br>
I am currently on the drug as well via the extended study (PIII is finished) but unfortunately, I have yet to notice a major difference... However, I am pretty certain I was on placebo first, and maybe this will take a few months to work its magic. Who knows.
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
I am currently on the drug as well via the extended study (PIII is finished) but unfortunately, I have yet to notice a major difference... However, I am pretty certain I was on placebo first, and maybe this will take a few months to work its magic. Who knows.
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
I am currently on the drug as well via the extended study (PIII is finished) but unfortunately, I have yet to notice a major difference... However, I am pretty certain I was on placebo first, and maybe this will take a few months to work its magic. Who knows.
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
The positive is that these drugs ARE coming out, so even if PTC doesn't nail it - we are on the right track <img src="i/expressions/face-icon-small-smile.gif" border="0">
lisayourman
New member
I have two young adults on the trial. It has a very intense treatment plan and whether it is ever approved by the FDA, in addition to your normal routine of CPT, inhaled medications, oral medications, pancreatic enzymes, regular antibiotics, IV's and bacterias, Ataluren will be an addition to the treatment plan. The treatment for Ataluren is a powder mixed with water three times per day and massive amounts of water to flush the kidneys. This form (powder) is how it will be approved by the FDA. Not an easy treatment plan to lead a normal life.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.
lisayourman
New member
I have two young adults on the trial. It has a very intense treatment plan and whether it is ever approved by the FDA, in addition to your normal routine of CPT, inhaled medications, oral medications, pancreatic enzymes, regular antibiotics, IV's and bacterias, Ataluren will be an addition to the treatment plan. The treatment for Ataluren is a powder mixed with water three times per day and massive amounts of water to flush the kidneys. This form (powder) is how it will be approved by the FDA. Not an easy treatment plan to lead a normal life.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.
lisayourman
New member
I have two young adults on the trial. It has a very intense treatment plan and whether it is ever approved by the FDA, in addition to your normal routine of CPT, inhaled medications, oral medications, pancreatic enzymes, regular antibiotics, IV's and bacterias, Ataluren will be an addition to the treatment plan. The treatment for Ataluren is a powder mixed with water three times per day and massive amounts of water to flush the kidneys. This form (powder) is how it will be approved by the FDA. Not an easy treatment plan to lead a normal life.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.
No change to FEV1. Disappointed in the silence of the kidney issues across the patients enrolled. Disappointed that the consent is not being followed. Disappointed that there is nothing about the inhalation of the powder through the sinuses and lungs of patients and parents when opening the sachets.