It took some wrangling, a temper tantrum or two, major Mama Bear Mode, tons of patience and lots of faxing—who faxes anymore? —but it has finally arrived, Emlen’s first box of TRIKAFTA.
TRIKAFTA targets the underlying cause of CF; it does not just treat the symptoms. Once Emlen is on the therapy his lung function should increase. His GI and malabsorption issues should ease. He should have less exacerbations and infections. Colds should not render him useless for weeks at a time. He should have more energy—heaven help us. And, most importantly, his life expectancy should increase.
All with just three pills a day, two in the morning and one in the evening.
We still have to keep up with all of Emlen’s daily therapies as this is not a cure, but it is a huge step in the right direction for longevity.
I feel like I just drank a gallon of sap from a 1000-year-old pine tree.
Many, many thanks to all of you who responded with your thoughts and support over the last week. You, our friends and family, are the best…
Here are the greatest hits:
From Hillary, swim mom, family friend and Emlen’s swim coach:
I am reading this email...and crying. It is an immense relief to hear that research is successfully moving forward. I just cannot imagine your daily struggle, and to know that it may be a tad less from here on out is the best news I’ve ever been privy to. I love Emlen and admire your family in countless ways. Thank you for sharing this amazing news!!!!!
From Chris, brother-in-law and CF Cycle for Life participant—every year:
Elizabeth, we were so excited to hear this news!!! Thanks for the update. I hope the results are even better than expected!
From Rika, swim mom and family friend:
This is such GREAT news and brings tears to my eyes so I can only imagine how you are feeling. I did not dare ask when I read the article in the paper yesterday if Emlen would be part of the group this helped, although 90% is a huge number, someone has to be in the 10% I suppose.
There are no other words then YEAH!!!! Everyone in my family will be thrilled. I look forward to sharing the news with them.
From Baba, Emlen’s grandmother:
I am moved to tears. Thank God for Vertex. Please let us know when he starts taking it and if you notice a difference.
From Erik, family friend and LLR webmaster extraordinaire:
Fantastic News. Happy for you, and this has lifted my spirit for the day.
Raising a shot glass of sap.
From Ellen, family friend:
Oh my God! What an amazing piece of news! I am overjoyed!
From Grammy, Emlen’s grandmother:
FABULOUS! What welcome and happy news you have received!
From Mary, Emlen’s middle school nurse:
This is such wonderful and hopeful news! I am beyond thrilled that it was made available early and that Emlen will be able to start right away.
Cheers on the pine sap, I'll have a cup with you!
From Aunt Emily, sister-in-law:
This is fantastic news! Thanks, so much, for keeping us in the loop. We’re hopeful that this will be a HUGE improvement for Emlen.
You and Christian are such strong, wonderful parents and Emlen is such a trooper. We’re in awe of his positivity and strength as he manages all that CF brings on.
Sending lots of love to all four of you and keeping Emlen in our prayers as he begins this new step in treatment.
From Grandy, Emlen’s grandfather:
This is indeed good news!
From Melissa, family friend:
Oh, my goodness! I had no idea when I read about this that it helps Emlen! That is amazing! I'm so happy for him! Sap all around!!!!!
From the Hesbach Family, ex-pat family friends:
That is amazing. We are so happy for you all. Lots of positive vibes from across the ocean!
Tom, Amie, and Siena
From Meg, Emlen’s elementary school nurse:
I heard rumors....
I have tears in my eyes after reading this...
Thank you for sharing with me, I miss my friend.
How is the year going??
From Tilda, my godmother:
Keep the faith
Onwards and upwards
From Aunt Lindsey, my aunt.
We are so happy for you all. Leigh and I just got back from a birthday celebration in Morro Bay (mine, and 70, gulp!), and I read about Trikafta from a CF new release. That is fantastic news. We send you all our love, and to Henry a special woof from Cozy (who also turned 70).
From Uncle Ted, stepbrother:
Wow! What an update. Brings a smile to hear there is something that can do more for him than just treat the symptoms. Keep up the great work supporting that amazing kid! Proud of you two as well as him!
From Carol, family friend and neighbor:
Oh my goodness!!! That is just fabulous news!! I'm so thrilled for all of you!!! Woohoo! I'll assume that there are no or very low/little side effects? But it's so nice to hear that for once the FDA does something in a timely fashion - there are so many ridiculous delays. Big cheers & hugs to all 4 of you!!
From Alison, cousin:
This is such incredible news to hear! We are so excited for Emlen and the whole family about all of the positive steps to come. Fingers crossed Emlen's well check goes well tomorrow and the pills land in your hands very soon. Sending good thoughts for Henry's challenges too.
How lucky the boys are to have you and Christian. I can only imagine what a difficult road it's been.
Sending hugs to everyone.
From Serena, family friend and Erik’s wife:
This is such happy news Elizabeth! Thank you so much for sharing it with us. We are sending our well wishes, for Emlen, and all of you. If there is anything, we can do to support you with this, please let us know.
We love you guys and hope we can meet up to celebrate with you at a lake in NH sometime soon.
From Sarah, swim mom and family friend:
This is amazing news. Thank you for sharing and keeping us in the loop. I had wondered if this development was applicable to Emlen when I heard it on the news recently.
Our continued thoughts are with you guys.
From Uncle Kurt, college roommate, family friend and godfather to Henry and Emlen:
Stellar news, unless of course you're a Christian Scientist. Go team pharma! Love and miss you guys. Tell Xian to give me a call sometime. I need parenting advice. It's gonna be a girl.
From Peter, Emlen’s grandfather:
Hi Elizabeth, that’s wonderful news and we are happy to know that Emlen’s risks and discomfort will improve, hopefully significantly. We are both fine and enjoying life together, but it is hard not to worry about our fair state with 90-degree weather in late October. Hello to all the family. Peter
From Aunt Cheryl, stepsister:
Amazing news, Elizabeth. So glad TRIKAFTA is out and available. We are all thrilled for Emlen and for all of you.
Fingers crossed for way fewer hospital visits in years to come.
They were all great. Every single response. I loved them all. I just could not include each and every one, otherwise this entry would go on forever. You know I have a problem with blog posts going on forever…
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