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2.5 years after this study was started, they FINALLY opened it up to those with rare mutations other than nonsense mutations. https://www.cff.org/Trials/finder/details/477/Rare-mutation-cell-collection-RARE...

Hello- These 2 websites will give you general and specific info. about different mutations. https://www.cff.org/What-is-CF/Genetics/Find-Out-More-About-Your-Mutations/ https://www.cftr2.org

shay, I apologize if my tone was harsh. I didn’t mean to undermine your knowledge or experience. This particular topic is an extremely emotionally charged one for me. Helping those with rare mutations gain access to these breakthrough therapies has been a 5 year long plight for me...

In response to above post from shay: Unlike nonsense mutations, the science for effective drugs for many of the rare missense mutations is there, but the FDA approval process hasn’t caught up with the science. I have a missense mutation and I’ve been on off-label Kalydeco for 5 years. My...

:) Great article about the triple combo, Trikafta: https://www.washingtonpost.com/health/2019/10/31/long-awaited-cystic-fibrosis-drug-could-turn-deadly-disease-into-manageable-condition/

I just sent the webinar link to your Private Messages. Let me know if you didn't receive it. I frequently have problems with private messages not being sent or received.

The Cystic Fibrosis Reproductive and Sexual Health Collaborative (CFReSHC) hosted a webinar by Dr. Jennifer Taylor-Cousar on CFTR modulators and your Sexual Health back in January. Modulators and pregnancy and lactation was one of the topics. Check your personal messages for the link to the...

There's also a facebook group that you can join where people have been sharing their experiences with VX-445 Expanded Access https://www.facebook.com/groups/vx445compassionateuse/

FYI- There's an expanded access program (EAP) compassionate use program available for the new triple combo, VX-445 As far as I know, it's not limited to US patients. (I think Ireland and UK are included, but other countries may be eligible). It's difficult to get any information from...

Here’s a website with different financial assistance programs for CF: https://www.needymeds.org/copay_diseases.taf?_function=summary&disease_eng=Cystic%20Fibrosis No catastrophic fund for Louisiana that I could find. This is a very old document from 2008 that outlines different state funding...

Here’s a website with different financial assistance programs for CF: https://www.needymeds.org/copay_diseases.taf?_function=summary&disease_eng=Cystic%20Fibrosis No catastrophic fund for Louisiana that I could find. This is a very old document from 2008 that outlines different state funding...

I just thought of something else. I'm not sure if you already know about the Healthwell Foundation. I would think they also have the off-label restriction, but it's worth looking into: https://www.healthwellfoundation.org/fund/cystic-fibrosis-treatments-2/

Unfortunately, you won't be able to get co-pay assistance for off-label, it's not legally permitted. I know of others in your situation, some who don’t have an out-of-pocket max so their co-pays are exhausting their retirement savings; some have had to stop taking it all together because they...

Unfortunately, you won't be able to get co-pay assistance for off-label, it's not legally permitted. I know of others in your situation, some who don’t have an out-of-pocket max so their co-pays are exhausting their retirement savings; some have had to stop taking it all together because they...

When they have the 24 week data in, they'll choose which triple they’re going to submit for new drug application. Sounds like it will be sometime in third quarter of 2019. FDA will hopefully accept priority review of application and they’ll set an action date to review. Because it will be a...

I might not have this right, but I’ve been given the impression that Vertex will attempt to get triple combo approved for anyone with at least one df508 mutation, even if their other mutation wasn’t included in the clinical trials of triple combo. I’m not sure why they didn’t have a separate...

Very exciting results announced today for VX-445 :) Sounds like they'll review more data at end of 24 weeks and decide between VX-659 and VX-445 From Vertex press release on 3/6...

https://www.foundcare.com/Patients/everyday-specials.html I now have to use Accredo for Cayston, but used to get it filled through Foundation Care and got hypertonic saline for free for years, so I think it's a long-term promotion. I still use FC for other prescriptions. They recently have...

Hi Matthew. Sorry to hear you're feeling alone. CF can be such an isolating disease. There's a CF discord where you'll be able to connect with others with CF that can relate to what you're feeling. I'm not a member, but heard positive things about it. "CF Discord is a place where you can...

Hi Kenna, Sorry to hear about the hemoptysis. I’m not sure if you have the space or the budget for this, but thought walking on a treadmill might be an option. Walking at a slow pace for longer periods wouldn’t increase heart rate too much, but would still give you some exercise. Some have...