Skip to main content

Exciting news

Have you heard the news??

Press release

Now you have :)

Every time I read this article, I want to jump up and down. Phase 2 of the VX-809 and Kalydeco study was successful in patients with deltaF508 mutation, the most common mutation that causes Cystic Fibrosis! (Actually, roughly 90% of CF patients have at least one copy of deltaF508.) In case you didn't know, studies testing new medicines go through three major phases. Phase 1 often screens for the safety of the medication and is usually tested in a small group. Phase 2 is a bit larger and tests the effectiveness and safety of the drug. Phase 3 is the final stage before approval from the FDA. In a Phase 3 trial, larger groups of people participate in it to affirm the drugs effectiveness, decide on common side effects, and figure out the best way to provide the drug to the public market. If a drug makes it through all three phases, it is sent to the FDA for the official stamp of approval. This can be a rather long process, often taking months or even years from the time the phase 3 trial is complete to when the drug is released to the pharmacies.

However, I am still thrilled with the results from this study. I have one copy of a deltaF508 mutation and one more rare mutation. Therefore, I could potentially be part of the Phase 3 study! It would mean so much to me to be part of this study. For starters, I would make a huge difference in the CF community. As well, I personally could receive huge benefits from this medicine. I could gain weight, increase my lung function, and maybe, just maybe, know what it feels like to inhale deeply! I'm going to try my best to get into a study somewhere, even if it means driving all over the country.

We live in exciting times in the CF community, my friends. I can't wait to see what God has in store for the science community in the near future!


  1. OMgosh, this is so exciting Emily!! I feel so happy for you!! =D


Post a Comment

Popular posts from this blog

Exciting news!!

It's been awhile since I've written a blog post. This semester has been busy--not only because of school stuff, but also because of exciting life things. About a month ago, the most amazing man got down on one knee and asked me to marry him! And of course, I said yes!

Honestly, there were times that I questioned if I would ever marry someone. Living with cystic fibrosis is hard. Choosing to be with someone with cystic fibrosis is almost crazy. Think about it; I am not a normal 23 year old. I have to plan and plan and plan to make sure I fit breathing treatments, exercise, and eating into my schedule. When I travel, I have to take a crazy amount of stuff with me--my Vest, nebulizers, compressor, pills, inhaled medication, puffers, and snacks. I have to make sure I sleep 8-9 hours a night because my body uses more energy than most, and I need sleep to fight infection. I have to have a course of IVs at least twice a year. It's hard to be spontaneous and adventurous because CF…

CF limits

I was always told I could do anything.

That CF couldn't stop me.

That, even though my day to day life looked a little different with treatments and pills and hospitalizations, I could still be "normal".

I'm finding out now that's not necessarily the case.

Growing up, I knew I was different, but I still functioned like a normal kid. The only time I remember CF limiting me was my freshman and sophomore years in high school. My doctor, mom, and I made the decision to sit out of marching band my freshman year and to keep me on the sidelines running the metronome and helping how I could without actually participating my sophomore year. Junior year I was finally able to join marching band, and my senior year I was a drum major, so CF didn't limit me that much by the end of it all. I finished college in four years with a major, a minor, honors, and summa cum laude. I am in grad school now and will graduate on time summa cum laude with my masters in speech pathology.…

The false narrative

Today I was at church with my parents. After the baby dedication, the pastor prayed over the families. It was a fine prayer until he said something along the lines of "raising kids in a Christian home is the best way to ensure kids grow up healthy". This is when I opened my eyes and tuned out the rest of the prayer. Honestly, this is where I tuned out the rest of the service. This false narrative is exactly why American Christianity can be so out of touch with the world.
No. No. No. This is not how God works. Yes, in a world without struggle and pain and heartache, I wouldn't have cystic fibrosis. But in our current, broken world God uses illness and weakness to prove His strength and power and love. If God wanted to heal me, I have full confidence that He could and that He would. I know there are people who have experienced divine healing. But in many cases, God uses our weaknesses rather than spontaneously healing us. In 2 Corinthians 12, Paul says, "But He (the Lo…