tag:blogger.com,1999:blog-7535774120903384819.post2046641921231408176..comments2023-12-27T00:26:40.602-08:00Comments on Possibilities: Gating Mutations and Kalydeco...A summary from the NACFCRebeccahttp://www.blogger.com/profile/01834134399653596127noreply@blogger.comBlogger5125tag:blogger.com,1999:blog-7535774120903384819.post-45195589598613829152013-03-27T21:14:24.501-07:002013-03-27T21:14:24.501-07:00Your article was beyond helpful! I'm a mom of ...Your article was beyond helpful! I'm a mom of a CFer, who's 10. I'm just now realizing what great news this new drug is. My son has a double S549N gene mutation. That is one of the gating mutations that Kalydco may be effective with. My heart is beating so fast right now to think that there may be something out there that could help him?!! Your blog has made me understand so much more than I ever have. Watch out… I'm on a path!! Thank you , thank you, thank you!!!<br />OC 2010https://www.blogger.com/profile/10555831368972937134noreply@blogger.comtag:blogger.com,1999:blog-7535774120903384819.post-55395666836512194932012-02-09T23:32:05.779-08:002012-02-09T23:32:05.779-08:00No details about the European trials. And you are...No details about the European trials. And you are absolutely correct in saying that VX-770 potentiates all those other gating mutations "as well or better"! I can't describe how positive I feel about the chances for other gating mutations to get this drug soon. On the last point, also right about sweat chloride. Not all patients dropped below 59 mmols. However, those patients still exhibited the positive clinical benefits of increased lung function, reduced exacerbations, etc... Thanks for reading!Rebeccahttps://www.blogger.com/profile/01834134399653596127noreply@blogger.comtag:blogger.com,1999:blog-7535774120903384819.post-81649437102233966892012-02-08T16:43:35.145-08:002012-02-08T16:43:35.145-08:00Do you have any idea about trial locations (in Eur...Do you have any idea about trial locations (in Europe) for the upcoming 2-5 year olds for VX-770?<br /><br />Btw, I notice the open channel probability for GD551D are quite low compared to the other gating mutations. Only 10-15% of the time? I also noticed that avg salt levels in the P3 sweat test were about 50mmols. What is the error bar on the lower/upper side? Were there some patients with sweat tests above the dreaded 59mmols, and, therefore, not 'cured'? Is VX-770 dose-dependent - can you increase the dose to increase the channel open probability?<br /><br />ThanksJim Dowlinghttps://www.blogger.com/profile/06334659492165169076noreply@blogger.comtag:blogger.com,1999:blog-7535774120903384819.post-58542809569496473212012-02-08T04:42:20.306-08:002012-02-08T04:42:20.306-08:00Prayers and hope to all....xox Laura and Graham...Prayers and hope to all....xox Laura and Graham's bright spiritAnonymousnoreply@blogger.comtag:blogger.com,1999:blog-7535774120903384819.post-47974830395684492942012-02-07T22:48:08.614-08:002012-02-07T22:48:08.614-08:00This is super interesting, Rebecca. Thank you!!This is super interesting, Rebecca. Thank you!!BGhttps://www.blogger.com/profile/06373281991796003525noreply@blogger.com