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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.
...

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...

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...

:) 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...

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...

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...

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...

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:
...

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...

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...

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...

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...

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...

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. ...

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...

For those who don't already subscribe to the CF Roundtable newsletter:

The special winter CF Roundtable® issue on Organ Transplantation in CF will be out soon.

Subscribe by January 26th to...

Novartis Patient Assistance (the makers of Tobi) might be able to help:
https://www.patientassistancenow.com/

CFF Compass is another resource:...

I’m so sorry to hear of your boyfriend’s relapse of pancreatic cancer. It sounds like a perfect idea to plan a relaxing trip for him.

Take a Breather Foundation is a wonderful organization that...

Beth Sufian is Director of the CF Legal Information Hotline. She was the lawyer interviewed in the article/video clip.

CF Legal Information Hotline

1-800-622-0385. All contacts are free and...

I'm guessing most have seen this already, but thought I'd share in case you missed it:

Incredible Phase 3 results for VX-659!!!:)

From Vertex Press Release on 11/27:

Two Phase 3 Studies of...