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We were in a similar situation with our second child, but decided that the test which increases the chances of a miscarriage was not worth our peace of mind. When were were considering it, the increased chance of miscarriage was greater than now, but any increase would steer me away from this...
Not sure about other online support, but we went through something similar with my mother-in-law with a brain injury. We ended up going with home health care paid for by Medicare. Although it is a pain at times, we avoided the nightmarish Alzheimer nursing homes.
Talked with Vertex, they said they are still going to pursue FDA approval for Kalydeco. I asked a ton of questions but failed to ask how they were going to proceed and the time frame. I will let you know when I find out more.
You might want to focus on the known gene first N1303K. Typically, outcomes are tied more to their best functioning gene. If the other operates like D508 it would likely be the worst functioning gene. For example, I have D508 but my other gene is normal so I am only a carrier without symptoms.
Cell correction vs. reversing damage.
This is an excellent point. Kayladeco will not fix the damage from CF over years. It will fix the cause of the damage. Any immediate increase in lung function or pancreas function is due allowing the remaining healthy parts to function better. Just as...
Found a report that said the study failed?!!! Typical, the business people read the first line of a technical report and made false conclusions. The drug worked and still failed the primary endpoint.
Technical people need to be careful when defining a study so that it has a chance to show...
More related info to the Vertex (Kalydeco) Ivacaftor R117H study
Did some additional research. I found a study of G551D with kids less than 12. Those kids had a mean baseline FEV1 of 84.2 ± 18.07 This is 11 percent worse than the kids in the R117H study. Not...
Is it just me or was this study poorly defined, providing misleading results?
I understand this was a difficult study to perform with so few participants, but it should have been tailored to the population.
They took patients under 12 who had FEV1 between 40 - 105% and patients above that...
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