CF is horrible. I had no idea that there was a new treatment and that it was so effective.
Has a double lung transplant in 2007. My CF doctor told me today he doesn’t even really refer patients to transplant centers anymore because of the newest drugs.
It really is amazing. When I had my transplant , the post surgery rehab was walking on treadmills and light weights. My transplant rehab office was 60+ year olds and 20 year olds with CF, nothing really in between.
It’s awesome because the extra effect is that the lungs that CF patients would get can no goto another patient that needs them.
The idea of being completely cleaned out of mucus like that sounds wonderful, i haven't felt like i could breath perfectly since i was like, a teenager
I would be seriously tempted to try this stuff as someone who doesn't suffer from CF just to see what it would be like.
That being said, I'm sure the side effects for people who do not have an illness are probably pretty horrific. I have not done any research, but usually very powerful drugs have very powerful side effects, especially for people who won't see any substantial benefit for the drug.
I'm told my family medical history is pretty unpleasant, to the degree i have been apologized to for genetics being passed down; unfortunately getting any specifics beyond "diabetes and misc. heart issues", and some vague symptoms, is like pulling teeth. Some of the symptoms of CF are.... alarmingly familiar. Mainly thick mucus, frequent sinus infections, inflamed nose, difficulty breathing, etc
Edit: oh, and most of my extended family is dead. I've got less than 10 living biological relatives that im aware of
It targets a very specific genetic mutation, so it does nothing for people who don't have that mutation (including other people with CF).
Beyond the pricing issue you already found, unless you have specific genetic mutations that cause CF like the F508del mutation, these drugs won’t really do anything for you.
Am I the only one asking how they know they will live into their seventies if the drug was just approved in 2019?
The drug was likely out in human trials for years before it was approved, and they likely could see the recovery was commensurate with a normal human lifespan.
it's a good question - but it may be the expectation to "live a normal lifetime" or maybe people who participated in early trials? Don't know.
well there's still five or ten years where people will be able to live on earth.
i recently listened to a podcast with a guest who has CF, and she described how her life changed after she took this medication during the trial period. she said she would probably be dead by now if she hadn't had it.
go on to live into their seventies
oh. they were like 65 years old already?
Lost my cousin to CF in the 90s, he was just 13 years old. It was a horrible condition with a lifetime of being in and out of the hospital, many hours long coughing fits, and highly invasive interventions. I'm really happy to see that progress at this level is being made.
That was the most disgusting news of medical awe I've read this year.
Is it gene therapy? That was the last time I heard about a new cystic fibrosis treatment.
