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Truly a modern day miracle delivered to the largest CF community. Hopefully this gets through the FDA/Government agencies/insurance providers in a timely manner. Hats off to the CF Foundation and Vertex. Seems like most people fail to realize that this type of treatment basically plugs the...

Last thing I heard was that Vertex and the Canadian government were in negotiations on what to charge for the drug. I am guessing that is holding up everyone in Canada from getting the drug unless you are in a study? Pretty crazy that a cure/treatment is finally discovered and governments/drug...

We are in the same boat with R117H and DF508. Looks like the FDA should approve for use over 18, but the insurance denied the off label use, stating that the FDA has not yet approved the med for my son's genotype. He coughed most of last night. Seems like the process should take less than 9...

Country Price Australia Estimated USD $300,000 a year Canada At least USD $300,000 per year China No Information Available Germany No Information Available Japan No Information Available UK Estimated £182,625 per year USA $294,000 per year Mexico No...

Too much times two :) I would like to know what the costs are for Kalydeco in the countries with socialized medicine. I am guessing it is not 300k/year.

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Statistics As excited as I am about having new drugs enter the market, I know enough about statistics to know that using test sizes of < 100 of nearly anything with even a small amount of variation is not statistically significant. That said you can only work with what you have available...

Yes, I had heard that SC was not correlating to clinical benefit. Maybe it is due to the clinical benefit they were looking for had to be seen within the short trial period. I wonder how much variation in three months is typical for FEV1. My son's FEV1 went from about 91 all the way up to...

At first, I was a little confused about the results from this 4 week study. Here is my summary of what happened. They took people who were already taking Kalydeco and then also gave some of them VX-661. These people had one gene that was DelF508 and the other gene was one of the G551D...

My understanding is that Kalydeco will help in other areas (like the Pancreas) not just the lungs. The transplanted lungs are not made from CF cells so their would be no benefit for them directly.

A few comments, about the following. Long term damage reversal after using Kalydeco has not really been tested since it is relatively new. I would like to hear about the people using Kalydeco to see if anyone has increased their FEV1 after the initial improvement. The lungs are pretty...

Really enjoyed these comments above.

Many patients with R117H and Df508 have a reduced set of symptoms. How reduced depends on their variant (5T 7T or 9T). Please see the following link for more details. http://www.cftr2.org/r117h.php Also depends on the following TG tracs...

This makes sense unless the newborn screening comes back with the two known CF genes.

My wife and I are both carriers R117H/DF508, although I am not sure which variant of R117H. Anyway, one kid has be DX with CF other is a carrier with R117H. Carrier(only R117H), has worse pulminary function (PFT's) than the kid with CF, due to asthma. It is pretty common for carriers to have...

Pretty cool even if it was only done in a test tube. One step closer.

I really like yoga, but I am not sure it is ideal to help your lungs, since it is not very aerobic. That said it does wonders for your muscles and joints as you age, also it helps relieve stress which is important to overall health. Lastly, it does help with deep breathing which might help...

Good news for approx. 150 people with CF 6 and older. a supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene...

The only thing I can add is that the "del" is short for deletion in the dna. Typically del type mutations behave similarly. So maybe this means that the unknown one behaves similarly to a delta508 and that the combination of the two gene mutations would be similar to a person having two df508...

Newborn screening I was surprised that your child did not have a genetic screening test. I guess this varies with each state. Getting a genetic test is not only important for diagnosis, but also for treatment approach. Not all CF genetic mutations are currently treated the same way, as they...