So, please pardon any presumptiveness on my part but I've been brainstorming here re our ds. Basically, here's my thought process: Word on the street is that kids younger than 6 (as young as 2.5) are taking Kalydeco. Also, much off-label for other mutations, including some for double-deltas. So I've been pondering how to approach our doctor re trying with our son. My understanding is the that Phase 2 trial of 770 (Kalydeco) for double-deltas there was a small sub-set who benefits greatly from JUST 770. So why not try for ds to see if he might be one of those. But I think I'd have a hard sell because he is under 6 and right now am thinking to hold my fire until they approve for under 6. BUT then I thought of your daughter and though MAYBE you could get doctor to try and insurance might approve--if not straight out maybe for 30 days to see if any improvement. The research is published in April 2012 Chest (search for Ivacaftor sp? in pubmed).
As an investor, I have been focused on the financial side of Vertex's CF drugs but the the success of these medicines in treating CF patients is really the bigger story. It is what Vertex will be known for, rather than how much money they made. I can tell you from first hand experience during my residency training (which was at a hospital that is a CF center) what a devastating and heartbreaking disease this is. Regardless of the business success of Vertex, I hope these new medicines will work for your son and that the insurance companies will pay for off-label use so he can try them before all the studies and approvals are complete.
Thanks for your kind note. And I don't begrudge anyone a focus on the financial side--this is a financial message Board, not a CF support group. But I found such sollace here when dear son's diagnosis was too raw for me to hear all the heartbreak--whereas here I could hear/focus on the hope. And I owe a debt of gratitude to you and others (especially Thirdme, qdelfan, gladpick, tec and others) who took the time to explain things to me from a science perspective, so in return I am willing to share any of my insights. I also am a big believer in the free market system--businesses do good just by doing what they do best--innovate and in doing so help people/cure diseases--so I too want Vertex to succeed, because Vertex's success is our son's success!