I have CF, and it's great to see this market growing. The latest "wonder drug", Trikafta, has been a game changer.
* It eliminated most of my respiratory exacerbations - I used to have a hospital stay pretty much every spring, and haven't had any since starting it. (VERY timely, considering I started it just before the pandemic, and who knows what would have happened had I been admitted during that time)
* I also used to be underweight, and I now worry about losing weight (though gaining the weight during the pandemic was inconvenient, as for several months buying new clothes was tricky)
CF slowly irreparably damages various organs like the lungs and pancreas, and being 45, I'll never be "cured" as those systems have been effected. However, it's exciting to know that someone born with CF today may not ever experience that, but rather, just live a long normal life where they just take a few pills each day. (When I was born, I think the life expectancy was 7)
Obviously cost is an issue - it's VERY expensive. (retail $20k+ per MONTH) I have the best insurance my employer offers, and a great income, but many don't, and their copay assistance now caps out at $20k/year, so this can be a challenge. Many countries have better healthcare of course, but each new iteration of modulator (the category Trikafta falls into) tends to have a much longer approval cycle than in the US, with cost being a prime factor.
