Update: In January, Lance and I found out that Crosby is included in the group the FDA approved at the end of 2020 for the gene modulator Symdeko for certain rare CFTR mutations. We were in shock because while we have celebrated each and every drug approval, having rare mutations meant that up until now, Crosby was in the 10% of persons with CF that do not qualify for any of the ground-breaking drugs (Kaledeco, Orkambi, Symdeko, and Trikafta) that have been developed by the CF Foundation.
The new FDA approval only affected around 300 people and Crosby was one of them! If you have supported us through the Cystic Fibrosis Foundation in the past 7 years, THANK YOU. This is what we have been waiting for and your donations helped make this happen. With each drug approval, I would get emails or texts asking me if they benefited Crosby and I always had to say no, he was not included. But now, it’s HIS TURN.
The gene modulator medications for cystic fibrosis are
hailed as the greatest story in medicine today! They are literally transforming
a genetic disease. These drugs are not a treatment for the symptoms cystic
fibrosis causes, but are actual protein CORRECTORS. It is not a cure, but it is
as close as they have have ever gotten.
To give a brief description on how it works, Crosby’s salt chlorides
have never been able to come to his cells’ surfaces because they were blocked
by two gene mutations that screw up the salt and water content in his body.
This causes the mucus to thicken in his lungs and digestive system. Gene
modulators help the chlorides to come to the surface and virtually correct
this. If you want to learn more about how Symdeko works, here is a great video:
https://www.symdeko.com/how-symdeko-works
While we are so excited for Crosby to be eligible for
Symdeko, deciding to go ahead and have him take it was a tough decision. Since
insurance was a 3-month battle, that left a lot of time to research and weigh
out the choice. Reading over the good and the bad has caused a mix of emotions
for me.
There are side effects: Abdominal cramping, elevated liver
enzymes, and eye issues to name a few. One of my fears is that he won't be able
to continue the medication because of these side effects. I don’t want Crosby
to endure anymore pain in our pursuit of quality and quantity of life. He is
not too happy about the required blood tests every 3 months to make sure his
liver function is cooperating with the Symdeko. So I’m pretty anxious. This
medicine will change his body at a cellular level to work in a way that it
never has. That is a mighty decision to have make for someone else, and not one we've taken lightly. As his parents,
we want to protect Crosby but also not keep him from something that could be
beneficial for him.
All that said, we decided to start and I am excited to say
that on May 1st, the first day of Cystic Fibrosis Awareness Month,
Crosby took his first dose of Symdeko.
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The Cystic Fibrosis Foundation has promised that this is just the beginning. Kaledeco, Orkambi, Symdeko, and Trikafta will not be the last medical advancements for CF. Things can and will only get better.
I am so very grateful to Vertex, the Cystic Fibrosis
Foundation and everyone who made it possible for Crosby to get Symdeko. My
heart aches for the CF warriors who lost their lives to cystic fibrosis before
they ever had a chance to take these medicines. I continue to hold on to hope
for those who still aren’t eligible (even though the gap is narrowing.) My promise
is that I will continue to fight for and support the CF Foundation until all
people with CF have the transformative medicines they need… Until It’s Done!
Great Strides is on hold again for 2021 due to COVID-19
and needing to protect the CF community. BUT! We are still fundraising for the CF Foundation to keep the momentum going and
you can visit our page here: THE CROSBY SHOW
Love,
Lance, Carmen, and Crosby
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