New Drugs for CF

[rosesixtyfive]

Hi, I have a three month old who was diagnosed with CF at birth. I have been researching everything for him - maybe little too much research. I ran into this article, and the drug Miglustat sounds so promising for CF. Does anyone know why it is not mentioned on the CFF drug pipeline? Why aren't more people talking about it? I do think we are on the verge of some major breakthrough for all genetic diseases. You can read this article at this web site:
<a target=_blank class=ftalternatingbarlinklarge href="http://">http://insciences.org/article.php?article_id=6237</a>http://insciences.org/article.php?article_id=6237
Please let me know what you think.
rosesixtyfive, mother of Sam, double delta f508, and a fighter

 

[rosesixtyfive]

Hi, I have a three month old who was diagnosed with CF at birth. I have been researching everything for him - maybe little too much research. I ran into this article, and the drug Miglustat sounds so promising for CF. Does anyone know why it is not mentioned on the CFF drug pipeline? Why aren't more people talking about it? I do think we are on the verge of some major breakthrough for all genetic diseases. You can read this article at this web site:
<a target=_blank class=ftalternatingbarlinklarge href="http://">http://insciences.org/article.php?article_id=6237</a>http://insciences.org/article.php?article_id=6237
Please let me know what you think.
rosesixtyfive, mother of Sam, double delta f508, and a fighter

 

[rosesixtyfive]

Hi, I have a three month old who was diagnosed with CF at birth. I have been researching everything for him - maybe little too much research. I ran into this article, and the drug Miglustat sounds so promising for CF. Does anyone know why it is not mentioned on the CFF drug pipeline? Why aren't more people talking about it? I do think we are on the verge of some major breakthrough for all genetic diseases. You can read this article at this web site:
<a target=_blank class=ftalternatingbarlinklarge href="http://">http://insciences.org/article.php?article_id=6237</a>http://insciences.org/article.php?article_id=6237
Please let me know what you think.
rosesixtyfive, mother of Sam, double delta f508, and a fighter

 

[rosesixtyfive]

Hi, I have a three month old who was diagnosed with CF at birth. I have been researching everything for him - maybe little too much research. I ran into this article, and the drug Miglustat sounds so promising for CF. Does anyone know why it is not mentioned on the CFF drug pipeline? Why aren't more people talking about it? I do think we are on the verge of some major breakthrough for all genetic diseases. You can read this article at this web site:
<a target=_blank class=ftalternatingbarlinklarge href="http://">http://insciences.org/article.php?article_id=6237</a>http://insciences.org/article.php?article_id=6237
Please let me know what you think.
rosesixtyfive, mother of Sam, double delta f508, and a fighter

 

[rosesixtyfive]

Hi, I have a three month old who was diagnosed with CF at birth. I have been researching everything for him - maybe little too much research. I ran into this article, and the drug Miglustat sounds so promising for CF. Does anyone know why it is not mentioned on the CFF drug pipeline? Why aren't more people talking about it? I do think we are on the verge of some major breakthrough for all genetic diseases. You can read this article at this web site:
<br /> <a target=_blank class=ftalternatingbarlinklarge href="http://">http://insciences.org/article.php?article_id=6237</a>http://insciences.org/article.php?article_id=6237
<br />Please let me know what you think.
<br />rosesixtyfive, mother of Sam, double delta f508, and a fighter

 

[bet10]

Hi, I have a six month old and like you am researching madly. I have not heard about this, but wow, sounds exciting!! Hopefully others here can shed some light on this for us all. I wonder if that means you still have to do all the treatments, or is it some sort of magic pill? Fingers crossed...

Natalie, Mom to Tom w/cf (DDF508) and Jack, 3 no cf

 

[bet10]

Hi, I have a six month old and like you am researching madly. I have not heard about this, but wow, sounds exciting!! Hopefully others here can shed some light on this for us all. I wonder if that means you still have to do all the treatments, or is it some sort of magic pill? Fingers crossed...

Natalie, Mom to Tom w/cf (DDF508) and Jack, 3 no cf

 

[bet10]

Hi, I have a six month old and like you am researching madly. I have not heard about this, but wow, sounds exciting!! Hopefully others here can shed some light on this for us all. I wonder if that means you still have to do all the treatments, or is it some sort of magic pill? Fingers crossed...

Natalie, Mom to Tom w/cf (DDF508) and Jack, 3 no cf

 

[bet10]

Hi, I have a six month old and like you am researching madly. I have not heard about this, but wow, sounds exciting!! Hopefully others here can shed some light on this for us all. I wonder if that means you still have to do all the treatments, or is it some sort of magic pill? Fingers crossed...

Natalie, Mom to Tom w/cf (DDF508) and Jack, 3 no cf

 

[bet10]

Hi, I have a six month old and like you am researching madly. I have not heard about this, but wow, sounds exciting!! Hopefully others here can shed some light on this for us all. I wonder if that means you still have to do all the treatments, or is it some sort of magic pill? Fingers crossed...
<br />
<br />Natalie, Mom to Tom w/cf (DDF508) and Jack, 3 no cf

 

[hmw]

Any kind of 'magic pill' that will replace everything else necessary in treating this disease might be too much to hope for anytime soon, but several drugs are in the works that hopefully will be successful in addressing the cause of the disease, rather than the drugs we've had to date that can only address symptoms. I have no idea why this one isn't mentioned in the drug pipeline (unless it is by another name); based on what I was able to read the trial that was already completed only had 15 participants so hopefully the ongoing one is working with many more people and will get more conclusive evidence on what this drug can do.

Many drugs have reached various stages of development and don't work out for various reasons; it can be hard to know when to get excited about something/how much hope to pin on anything in particular. But anything that can correct the actual defects that cause the disease rather than just act as 'bandaids' for the symptoms are really worth investigating, as far as I am concerned!

Some information I could find about Miglustat...

<a target=_blank class=ftalternatingbarlinklarge href="http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page">About miglustat in clinical development for cystic fibrosis</a>

From July, 2009: <a target=_blank class=ftalternatingbarlinklarge href="http://www.news-medical.net/news/20090721/Miglustat-shows-promise-for-cystic-fibrosis.aspx">Miglustat shows promise for cystic fibrosis</a>

Studies in mice: <a target=_blank class=ftalternatingbarlinklarge href="http://ajrccm.atsjournals.org/cgi/content/abstract/179/11/1022">Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice</a>

Active Phase II Trial going on at this time in Europe: <a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct2/show/NCT00945347?term=miglustat&recr=Open&rank=1">Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?</a>

 

[hmw]

Any kind of 'magic pill' that will replace everything else necessary in treating this disease might be too much to hope for anytime soon, but several drugs are in the works that hopefully will be successful in addressing the cause of the disease, rather than the drugs we've had to date that can only address symptoms. I have no idea why this one isn't mentioned in the drug pipeline (unless it is by another name); based on what I was able to read the trial that was already completed only had 15 participants so hopefully the ongoing one is working with many more people and will get more conclusive evidence on what this drug can do.

Many drugs have reached various stages of development and don't work out for various reasons; it can be hard to know when to get excited about something/how much hope to pin on anything in particular. But anything that can correct the actual defects that cause the disease rather than just act as 'bandaids' for the symptoms are really worth investigating, as far as I am concerned!

Some information I could find about Miglustat...

<a target=_blank class=ftalternatingbarlinklarge href="http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page">About miglustat in clinical development for cystic fibrosis</a>

From July, 2009: <a target=_blank class=ftalternatingbarlinklarge href="http://www.news-medical.net/news/20090721/Miglustat-shows-promise-for-cystic-fibrosis.aspx">Miglustat shows promise for cystic fibrosis</a>

Studies in mice: <a target=_blank class=ftalternatingbarlinklarge href="http://ajrccm.atsjournals.org/cgi/content/abstract/179/11/1022">Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice</a>

Active Phase II Trial going on at this time in Europe: <a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct2/show/NCT00945347?term=miglustat&recr=Open&rank=1">Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?</a>

 

[hmw]

Any kind of 'magic pill' that will replace everything else necessary in treating this disease might be too much to hope for anytime soon, but several drugs are in the works that hopefully will be successful in addressing the cause of the disease, rather than the drugs we've had to date that can only address symptoms. I have no idea why this one isn't mentioned in the drug pipeline (unless it is by another name); based on what I was able to read the trial that was already completed only had 15 participants so hopefully the ongoing one is working with many more people and will get more conclusive evidence on what this drug can do.

Many drugs have reached various stages of development and don't work out for various reasons; it can be hard to know when to get excited about something/how much hope to pin on anything in particular. But anything that can correct the actual defects that cause the disease rather than just act as 'bandaids' for the symptoms are really worth investigating, as far as I am concerned!

Some information I could find about Miglustat...

<a target=_blank class=ftalternatingbarlinklarge href="http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page">About miglustat in clinical development for cystic fibrosis</a>

From July, 2009: <a target=_blank class=ftalternatingbarlinklarge href="http://www.news-medical.net/news/20090721/Miglustat-shows-promise-for-cystic-fibrosis.aspx">Miglustat shows promise for cystic fibrosis</a>

Studies in mice: <a target=_blank class=ftalternatingbarlinklarge href="http://ajrccm.atsjournals.org/cgi/content/abstract/179/11/1022">Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice</a>

Active Phase II Trial going on at this time in Europe: <a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct2/show/NCT00945347?term=miglustat&recr=Open&rank=1">Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?</a>

 

[hmw]

Any kind of 'magic pill' that will replace everything else necessary in treating this disease might be too much to hope for anytime soon, but several drugs are in the works that hopefully will be successful in addressing the cause of the disease, rather than the drugs we've had to date that can only address symptoms. I have no idea why this one isn't mentioned in the drug pipeline (unless it is by another name); based on what I was able to read the trial that was already completed only had 15 participants so hopefully the ongoing one is working with many more people and will get more conclusive evidence on what this drug can do.

Many drugs have reached various stages of development and don't work out for various reasons; it can be hard to know when to get excited about something/how much hope to pin on anything in particular. But anything that can correct the actual defects that cause the disease rather than just act as 'bandaids' for the symptoms are really worth investigating, as far as I am concerned!

Some information I could find about Miglustat...

<a target=_blank class=ftalternatingbarlinklarge href="http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page">About miglustat in clinical development for cystic fibrosis</a>

From July, 2009: <a target=_blank class=ftalternatingbarlinklarge href="http://www.news-medical.net/news/20090721/Miglustat-shows-promise-for-cystic-fibrosis.aspx">Miglustat shows promise for cystic fibrosis</a>

Studies in mice: <a target=_blank class=ftalternatingbarlinklarge href="http://ajrccm.atsjournals.org/cgi/content/abstract/179/11/1022">Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice</a>

Active Phase II Trial going on at this time in Europe: <a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct2/show/NCT00945347?term=miglustat&recr=Open&rank=1">Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?</a>

 

[hmw]

Any kind of 'magic pill' that will replace everything else necessary in treating this disease might be too much to hope for anytime soon, but several drugs are in the works that hopefully will be successful in addressing the cause of the disease, rather than the drugs we've had to date that can only address symptoms. I have no idea why this one isn't mentioned in the drug pipeline (unless it is by another name); based on what I was able to read the trial that was already completed only had 15 participants so hopefully the ongoing one is working with many more people and will get more conclusive evidence on what this drug can do.
<br />
<br />Many drugs have reached various stages of development and don't work out for various reasons; it can be hard to know when to get excited about something/how much hope to pin on anything in particular. But anything that can correct the actual defects that cause the disease rather than just act as 'bandaids' for the symptoms are really worth investigating, as far as I am concerned!
<br />
<br />Some information I could find about Miglustat...
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page">About miglustat in clinical development for cystic fibrosis</a>
<br />
<br />From July, 2009: <a target=_blank class=ftalternatingbarlinklarge href="http://www.news-medical.net/news/20090721/Miglustat-shows-promise-for-cystic-fibrosis.aspx">Miglustat shows promise for cystic fibrosis</a>
<br />
<br />Studies in mice: <a target=_blank class=ftalternatingbarlinklarge href="http://ajrccm.atsjournals.org/cgi/content/abstract/179/11/1022">Airway Delivery of Low-Dose Miglustat Normalizes Nasal Potential Difference in F508del Cystic Fibrosis Mice</a>
<br />
<br />Active Phase II Trial going on at this time in Europe: <a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct2/show/NCT00945347?term=miglustat&recr=Open&rank=1">Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?</a>

 

[just1more]

First before I reply, welcome to the site; I hope you can find a home here we have a great group of members.

That said:
I can't speak to this specific drug, however, optimism is great but often harder to find than realism in CF circles.

The primary reason it that we have members that have been told for the last 20 years that the cure was just around the corner.

There is NOTHING wrong with looking, reading and hoping for a cure. However, please guard your heart because nobody knows what will come tomorrow.

I personally feel that the greatest opportunity for CF is continued improvements to treatments that extend lifespan; not a 'cure'. The science behind curing any genetic disease is still in its infancy; whereas we have people here that are living testaments to the improvements in CF care that CFF has helped develop over the last 2 decades.

Just my .02 worth.

 

[just1more]

First before I reply, welcome to the site; I hope you can find a home here we have a great group of members.

That said:
I can't speak to this specific drug, however, optimism is great but often harder to find than realism in CF circles.

The primary reason it that we have members that have been told for the last 20 years that the cure was just around the corner.

There is NOTHING wrong with looking, reading and hoping for a cure. However, please guard your heart because nobody knows what will come tomorrow.

I personally feel that the greatest opportunity for CF is continued improvements to treatments that extend lifespan; not a 'cure'. The science behind curing any genetic disease is still in its infancy; whereas we have people here that are living testaments to the improvements in CF care that CFF has helped develop over the last 2 decades.

Just my .02 worth.

 

[just1more]

First before I reply, welcome to the site; I hope you can find a home here we have a great group of members.

That said:
I can't speak to this specific drug, however, optimism is great but often harder to find than realism in CF circles.

The primary reason it that we have members that have been told for the last 20 years that the cure was just around the corner.

There is NOTHING wrong with looking, reading and hoping for a cure. However, please guard your heart because nobody knows what will come tomorrow.

I personally feel that the greatest opportunity for CF is continued improvements to treatments that extend lifespan; not a 'cure'. The science behind curing any genetic disease is still in its infancy; whereas we have people here that are living testaments to the improvements in CF care that CFF has helped develop over the last 2 decades.

Just my .02 worth.

 

[just1more]

First before I reply, welcome to the site; I hope you can find a home here we have a great group of members.

That said:
I can't speak to this specific drug, however, optimism is great but often harder to find than realism in CF circles.

The primary reason it that we have members that have been told for the last 20 years that the cure was just around the corner.

There is NOTHING wrong with looking, reading and hoping for a cure. However, please guard your heart because nobody knows what will come tomorrow.

I personally feel that the greatest opportunity for CF is continued improvements to treatments that extend lifespan; not a 'cure'. The science behind curing any genetic disease is still in its infancy; whereas we have people here that are living testaments to the improvements in CF care that CFF has helped develop over the last 2 decades.

Just my .02 worth.

 

[just1more]

First before I reply, welcome to the site; I hope you can find a home here we have a great group of members.
<br />
<br />That said:
<br />I can't speak to this specific drug, however, optimism is great but often harder to find than realism in CF circles.
<br />
<br />The primary reason it that we have members that have been told for the last 20 years that the cure was just around the corner.
<br />
<br />There is NOTHING wrong with looking, reading and hoping for a cure. However, please guard your heart because nobody knows what will come tomorrow.
<br />
<br />I personally feel that the greatest opportunity for CF is continued improvements to treatments that extend lifespan; not a 'cure'. The science behind curing any genetic disease is still in its infancy; whereas we have people here that are living testaments to the improvements in CF care that CFF has helped develop over the last 2 decades.
<br />
<br />Just my .02 worth.