Today the FDA approved Kalydeco, a new drug that will help CF patients with the G551D mutation. You can read the article here.
It's a bittersweet moment. This drug will only help 4% of the CF population, and for those with this mutation, this is huge news for them: they will have an even better chance at living a normal life. For us, as it is for the remaining 96% of the population, it's still a waiting game. Since Judith does not carry a copy of this mutation, the drug won't help her.
Things are in the early stages of working on a drug for the DF508 mutation (which Judith has), but we don't know how long it will take for this to become available. It has to go through all of the courses. Maybe we'll get lucky and this drug will be released by the time Judith would be eligible to receive it.
I'm happy for the parents and children who will benefit from this drug - I truly am. At the same time, I wish we were the ones able to have the elation and hope for something that's as close to a cure as they've come so far.
No comments:
Post a Comment