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Posted

Wild.

 

At one point Both my sons were tested to rule out CF was a very scary time for us.

 

Thankfully they didnt have it and were diagnosed withsomething else but...............

Posted

I knew someone who died from CF, so this caught my eye. Thousands of families are devastated by this lung disease every year. This new drug stops the disease from progressing. Amazing stuff.

 

http://www.medpageto...cFibrosis/23973

 

This is the kind of story I like to read! Thanks for sharing Phantasm.

Posted

I was hoping the article was literally describing a "cure." it's a treatment far from a cure.

 

It's huge for CF patients.

 

When I was in college my girlfriend's sister had CF. Her name was Katherine. She had spent most of her life in and out of the hospital. Her mother had to spend hours every day pounding on her back to break up mucus so she could breathe. Every day. Just to keep her daughter alive.

 

They had dozens of medications but none of them did much.

 

Katherine died in May 1993 at the age of 18. I went to her funeral.

 

So, if it stops the disease from getting worse, it is life and death for these families.

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Posted

It's huge for CF patients.

 

When I was in college my girlfriend's sister had CF. Her name was Katherine. She had spent most of her life in and out of the hospital. Her mother had to spend hours every day pounding on her back to break up mucus so she could breathe. Every day. Just to keep her daughter alive.

 

They had dozens of medications but none of them did much.

 

Katherine died in May 1993 at the age of 18. I went to her funeral.

 

So, if it stops the disease from getting worse, it is life and death for these families.

 

Hey, I'm all for progress, and I too could share a story or two about CF patient tragedies and scientific sample assays I gathered and western blots that I had to do in my undergraduate internship at my Campus's Center of Comparative Respiratory Disease and Control, but what you have here Phantasm with that article is not the phenomenal "CURE" for CF patients that the title of this topic promises in the Tech/Sci Section.

 

Phantasm, please edit the topic title of this thread to properly reflect that this is a treatment used to remedy and ameliorate CF analogous to asthma sufferers relying on inhalers. This is far from a cure because nowhere does the article describe any methods to end/block/neutralize the hereditary genetic disorder from passing on to future offspring nor does it describe potentials to repair/infuse/replace the fault gene thus ending the mutation. The treatment cannot be considered curative because the CF mutation is still passed on genotypically. :hmm

Posted

Hey, I'm all for progress, and I too could share a story or two about CF patient tragedies and scientific sample assays I gathered and western blots that I had to do in my undergraduate internship at my Campus's Center of Comparative Respiratory Disease and Control, but what you have here Phantasm with that article is not the phenomenal "CURE" for CF patients that the title of this topic promises in the Tech/Sci Section.

 

Phantasm, please edit the topic title of this thread to properly reflect that this is a treatment used to remedy and ameliorate CF analogous to asthma sufferers relying on inhalers. This is far from a cure because nowhere does the article describe any methods to end/block/neutralize the hereditary genetic disorder from passing on to future offspring nor does it describe potentials to repair/infuse/replace the fault gene thus ending the mutation. The treatment cannot be considered curative because the CF mutation is still passed on genotypically. :hmm

 

Well, the gene therapy approach did not work, as you probably know, and therefore they came up with this drug.

 

They tried to use gene insertion to put healthy copies of the defective gene that causes CF into sick kids, and the kids died anyway, and actually died faster, because of side effects of having DNA randomly spliced throughout their chromosomes by a retrovirus.

 

There is no way to fix these defective genes at the time, I will admit that, but with this new drug these kids don't get any sicker. They improve lung fuction in most cases. As long as they take this medicine they should stay alive. It is very exciting.

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