ToniV 2.0: “Works as Designed”

Words Matter

A few people well acquainted with medication in general, and patients who have taken Orkambi since the studies began, have mentioned the symptoms I am experiencing are not “side effects” in the true sense of the word.

Let’s simplify this.

  • The Gene Mutation has caused a specific action within each cell of my body to not act correctly for 43 years.
  • I started to take the Medication, Orkambi.
  • The thick mucus is no longer being made.
  • Now, the mucus must get out somehow.

Continue reading

ToniV 2.0: The Orkambi Odyssey

Yesterday, I began taking the highly anticipated Cystic Fibrosis drug, Orkambi.

My box of hopes!

My box of hopes!

The medication, a combination of two drugs, aims to correct the defect that causes the characteristic thick mucus in CF patients with two copies of the most common gene mutation. The mutation, Delta f508, causes both lung and digestive involvement, in addition the sinuses, liver, and other organs. Continue reading

The Day of Reckoning

Tomorrow morning, I have my Pulmonary Function Test and some blood tests. A lot is riding on the results of these tests.

Providing my lung function is at least better than it was two weeks ago, and not going in the opposite direction, and my Liver function blood tests don’t show any irregular numbers, I am planning to finally begin taking the new Cystic Fibrosis drug, Orkambi. Continue reading

How Cystic Fibrosis Laughs at My Attempt to Plan Anything

In a way, it seems so long ago that I was last in the hospital, but it’s really only been four weeks.

Home! But…Healthy?

I completed my two-week course of my antibiotics exactly one month ago.

Normally after getting out of the hospital, I go through a period of recovery – still fighting the bacterial infections that landed me in there in the first place, and then trying to regain lost strength and energy from being laid out for so long. My body also has to recover from the effects of the strong antibiotics that, while killing off the bad bacteria in my lungs, also causes problems to other organs and systems.

I would experience a time when I felt GREAT (comparatively). This time, however, I missed out. Continue reading

Approved for New Cystic Fibrosis Medication!

I just wanted to share that I just got approved for the new Cystic Fibrosis drug that treats the root cause of the genetic defect of my mutation!!!

While taken, Orkambi corrects the defect that prevents the flow of salt and water from passing through cells properly, which creates the thick, sticky mucus in all of my organs, especially the lungs and digestive system. It is for CF patients with the mutation Delta f508, of which I carry two copies. It is also the most common mutation, and responsible for disease in most of my major organs.

While not a cure, this drug can potentially decrease the frequency and severity of complications, thus leading to a better quality of life. There are no guarantees, and there are side effects, but this is a HUGE chance for me – for us. My husband and I are excited and nervous that Orkambi will provide at least some benefits to increase quality of life.

I hope to share the process here. I see my doctors on Monday and hope to begin the medication in next week or so. If you’re interested in following my story, please subscribe for updates and you’ll be notified whenever I publish a new post.

Orkambi – Rejection, Appeal, and Life in Between

Here’s my first update since/about Orkambi, the new medication designed to improve the quality of life for a large percentage of Cystic Fibrosis patients.

My CF clinic sent the prescription to my Health Insurance provider, but they have rejected the pills based on need for proof that I…

A. Have CF,

B. Have the correct CF mutations,

C. The doctor prescribing the Orkambi is a CF specialist.

In the past two and a half weeks, there have been at least 10 phone calls and emails trying to move this process along. While I was on the fence in the beginning, unsure that I would take Orkambi, after getting sick again, and my doctor threatening to put me in the hospital a month ago, I started praying that I would be able to do everything possible to get approved.

I got through that exacerbation with 2 oral antibiotics. I was happy. I also saw my CF clinic and had my lung function tested – it was high enough that when I (presumably) started Orkambi ASAP, any side effects would be obvious, because I wasn’t sick at the time.

Now, as I watch my email inbox, make phone calls, and stalk my postman, I feel more and more that I want…I need…Orkambi.

But Wait, There’s More

I started to get sick again.

  • Productive nighttime cough
  • Fevers
  • Difficulty keeping up my oxygen saturation (I have no oxygen at home.) When asleep and after physically exertion, my oxygen saturation levels have dropped.
  • Walking up the stairs takes twice as long.

Now… It’s not emergent – I am not in respiratory distress, but they if they stay below 90 for any period of time, I will neeed oxygen.


If my symptoms collectively worsen, I will have to call my clinic and most likely will be placed in the hospital.


My fevers are relatively low, but still bothersome – and make me feel achy and tired!

Please pray, cross your fingers, or send your positive thoughts out into the universe that I have the courage and patience to do what I have to do should I go in the hospital, that my doctors have the wisdom make the best decisions possible, and that while I continue to fight for Orkambi, I will continue to hear all about all of the other CF patients who are taking the closest thing to gold we’ve ever had.

Until then….tell me about your Orkambi adventures, good and bad!!