Watching the results of Orkambi at the FDA- a big day for f508Del

12 05 2015

Vertex and FDA Advisory panel on a live blog.  Hard to say at this point where it is going, but the fact that a drug that targets f508del is even at the FDA is a step in the right direction.  If Orkambi worked like KALYDECO it would be so easy.   There are still lots of people for who Orkambi would be a major improvement to their quality of life.

Very interesting stuff.  We shall see what today brings.





Could tomorrow, be the start of more

11 05 2015

tomorrows than we ever dreamed possible?

Tomorrow. People with CF and their loved ones will be watching, listening and hoping. Tomorrow, the FDA will meet to evaluate the safety and effectiveness of a combination drug (Orkambi) and provide a recommendation for or against approval.

Tomorrow. The drug they are looking at tomorrow is targeted at people 12 and up with F508del homozygous cystic fibrosis (CF). This could directly impact Elina.  

Even if it isn’t this drug combination, the fact is we are close to a treatment that could dramatically change the course of this disease for OUR daughter.

This is what we are fighting for. It is right there, we are so close. The dollars you pledge, the money we are raising, it is making a difference. Please consider sponsoring us on our Great Strides walk, May 31, 2015.

gsw-lg-en

Tomorrows can be very exciting!  I am going to leave you with a little video from the American Cystic Fibrosis Foundation.  This video shows people with a different genetic mutation and shows their tomorrows on a drug called KALYDECO®.  There is hope that a drug like this will soon be available for Elina.

Thank you for sharing our journey.





Velo love

27 04 2015

There is something about seeing your kid ride a bike that just brings back the feeling of childhood.  We ride quite a bit, we ride for fun, for transportation and some days – just because we can.  We went on several bike rides today- Elina zipped along singing, laughing and ringing her bell.  Summer is coming and we are soooo ready for it. It is amazing how far a kid can ride while recovering from a pulmonary exacerbation!  What a rock star!

 She rides in style!  Streamers and all! I feel so lucky to love her and to be able to spend my days with her.  She has taught me everything. 





Lucky number 7?

26 04 2015

Tonight (yawn, stretch) this is how we are getting through the 7th nebulization of the day. Thankfully it is the last one of the day.   That works out to 4 different nebulizations, plus thumps in the morning before school and 3, plus thumps at night.  Each nebulization is 10-20 minutes long (depending on the volume of liquid and thumps are 20 min each session).  Everyday for a month.





One night at Nana’s

25 04 2015

Elina’s meds and sterilizer for one night at Nana’s.  Thankfully they have a compressor so we don’t need to pack that too! No wonder I often forget her toothbrush and toothpaste – good thing Nana always has one on hand. Lucky girl.  





my sleeping CF warrior

23 04 2015

 

Thumps (chest percussion therapy) puts Elina to sleep – yet even in her sleep this kid is breathing in TOBI like a champ!  CF never takes a break- so sometimes you have to kick CF in your sleep.

I don’t know how she does it!  But I sure do love her fight!





Gold!!!!

30 03 2015

Somethings money can’t buy.  Elina wanted to do Easter piñata for all boys and girls with toys inside.  What a lovely idea!  The following video is me explaining to her that we are not rich and so it will likely not happen this year.

http://youtu.be/oBp0HjlrbGU








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