Anyone with 3849+10kbc->t who is taking Kalydeco?

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CFSanDiego

New member
Hi. Is anyone with this mutation on Kalydeco off label yet? My 5yo is sick again and just looking for a little extra hope!

Thanks, Edan
 
C

cftrsplicing

New member
I know of 2 for sure.
One offlabel. One in the residual cftr function clinical trial.
No news at the moment. I ll report any news coming from the girl offlabel on cftrsplicing.com as soon as possible
 
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lifeisgood729

Guest
Edan,
I'm the "off label girl." I've been taking Kalydeco for a month now. Within a few hours I felt a difference in the amount of saliva in my mouth, and for a few days after, I was coughing out lots of junk because it was just so much easier to get it out. I repeated my PFTs after two weeks, and they increased from 1.74 to 1.83 liters FEV1. I never see any kind of increase these days, especially without antibiotics, so kalydeco is definitely working. I hope to see the numbers climb even higher as time goes by. For someone like me with significant lung damage, it still takes a lot of work and dedication to airway clearance, but kalydeco makes it possible to see immediate results from my airway clearance, as opposed to doing my Vest and seeing nothing move.

I will repeat my sweat test soon to see if my sweat chloride has decreased. It's only anecdotal, but when I sweat now, I no longer feel salt crystals on my forehead, so I'm expecting to see that my sweat chloride is lower. Overall, I'm excited about the potential of Kalydeco for 3849+10kb C-T, especially young kids with minimal lung damage. If the trials in Denver are successful, it might not be too long before our mutation is added to the FDA labeling.

Martha
 
C

CFSanDiego

New member
Oh my gosh Marha. I am so overwhelmingly happy for you that it sounds like it is working. I very much appreciate you takin the time to post about this. Please keep us up to date as you gain more insight re sweat test and so on.

.
 
P

pmotta

New member
This is such great news! My grandson (3) has the DF508 and 3819+10kbc>T. I've been following the forums for three years now, rarely post but love all the good information here. Joined the FB page! That was the best information yet! Thanks so much. Happy Holidays all!
 
Y

ymikhale

New member
lifeisgood729 said:
Edan,
I'm the "off label girl." I've been taking Kalydeco for a month now. Within a few hours I felt a difference in the amount of saliva in my mouth, and for a few days after, I was coughing out lots of junk because it was just so much easier to get it out. I repeated my PFTs after two weeks, and they increased from 1.74 to 1.83 liters FEV1. I never see any kind of increase these days, especially without antibiotics, so kalydeco is definitely working. I hope to see the numbers climb even higher as time goes by. For someone like me with significant lung damage, it still takes a lot of work and dedication to airway clearance, but kalydeco makes it possible to see immediate results from my airway clearance, as opposed to doing my Vest and seeing nothing move.

I will repeat my sweat test soon to see if my sweat chloride has decreased. It's only anecdotal, but when I sweat now, I no longer feel salt crystals on my forehead, so I'm expecting to see that my sweat chloride is lower. Overall, I'm excited about the potential of Kalydeco for 3849+10kb C-T, especially young kids with minimal lung damage. If the trials in Denver are successful, it might not be too long before our mutation is added to the FDA labeling.

Martha
Click to expand...
wow, I am soo glad i found this thread! my dd has this one too.
 
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