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Apparenty there are 141 registered CF patients worldwide with that mutation. This is out of 45000 CF patients registered. Pretty rare. It is a class 4 mutation which allows for the production of correct mRNA but limits the amount of it that can be active at the cell surface. But it is not so...

Apparenty there are 141 registered CF patients worldwide with that mutation. This is out of 45000 CF patients registered. Pretty rare. It is a class 4 mutation which allows for the production of correct mRNA but limits the amount of it that can be active at the cell surface. But it is not so...

Apparenty there are 141 registered CF patients worldwide with that mutation. This is out of 45000 CF patients registered. Pretty rare. It is a class 4 mutation which allows for the production of correct mRNA but limits the amount of it that can be active at the cell surface. But it is not so...

Thank you for linking that article that full set of information is the clearest I have seen all day.

Thank you for linking that article that full set of information is the clearest I have seen all day.

Thank you for linking that article that full set of information is the clearest I have seen all day.

Yes I am so hopeful but that stock drop surprised me. Could the investors and financial types have misinterpreted something? From what I read they gave 770 to some people but the therapy is meant for both drugs to be used and that only happened for some in the study and only then only for one...

Yes I am so hopeful but that stock drop surprised me. Could the investors and financial types have misinterpreted something? From what I read they gave 770 to some people but the therapy is meant for both drugs to be used and that only happened for some in the study and only then only for one...

Yes I am so hopeful but that stock drop surprised me. Could the investors and financial types have misinterpreted something? From what I read they gave 770 to some people but the therapy is meant for both drugs to be used and that only happened for some in the study and only then only for one...

Our daughter had no signs of CF other than a very mild reflux until a GI issue cropped up last summer and landed us with our diagnosis she was 9 years old. I wish you all the best and hope that you are able to get some definitive answers.

Our daughter had no signs of CF other than a very mild reflux until a GI issue cropped up last summer and landed us with our diagnosis she was 9 years old. I wish you all the best and hope that you are able to get some definitive answers.

Our daughter had no signs of CF other than a very mild reflux until a GI issue cropped up last summer and landed us with our diagnosis she was 9 years old. I wish you all the best and hope that you are able to get some definitive answers.

I think there is so little data on the rarer mutations like your second one and the variability among CFers is so great you cannot take chances. When our second one came with 2789+5 G>A I had been excited about the possibity of our child having a "mild" mutation. When I asked our doctor about...

I think there is so little data on the rarer mutations like your second one and the variability among CFers is so great you cannot take chances. When our second one came with 2789+5 G>A I had been excited about the possibity of our child having a "mild" mutation. When I asked our doctor about...

I think there is so little data on the rarer mutations like your second one and the variability among CFers is so great you cannot take chances. When our second one came with 2789+5 G>A I had been excited about the possibity of our child having a "mild" mutation. When I asked our doctor about...

We do 30 min on respretech preset but stopping every 5 min to "huff" so it really takes about 40 min. We do this twice a day and double up with pulmozyme in the evenings. Our little one is pancreatic insufficient but has no pulmonary issues so far we feel very lucky there since she is 9 and has...

We do 30 min on respretech preset but stopping every 5 min to "huff" so it really takes about 40 min. We do this twice a day and double up with pulmozyme in the evenings. Our little one is pancreatic insufficient but has no pulmonary issues so far we feel very lucky there since she is 9 and has...

We do 30 min on respretech preset but stopping every 5 min to "huff" so it really takes about 40 min. We do this twice a day and double up with pulmozyme in the evenings. Our little one is pancreatic insufficient but has no pulmonary issues so far we feel very lucky there since she is 9 and has...

Hello I have a question about the modulation therapies. If one of the VX trials shows effects for people with double F508 Del will this most likely benefit those with just one copy of F508 Del? Or do we have to wait for further studies? Which leads me to my second question, is one of a...

We have only been going for 6 months but we do love Hopkins and see Dr. Rosenstein.