Kalydeco question

[Hopeandlove]

My husband is going to start a trial on kalydeco next week. We are very excited but know he might get the placebo pill. I've been reading some things on here and it says the pill will not work on d508 mutation. Can someone explain this to me? Is it true? Is there anyone who has this gene that kalydeco did work for? Thank you for your help.

 

[FrancisB]

Kalydeco Alone

Kalydeco has not proven effective alone for the DF08 mutation, otherwise, the majority of people with CF would be prescribed it. However, if your husband has another mutation in addition to DF08, perhaps they are testing to see if it helps the other mutation. There are clinical studies ongoing now that are pairing Kalydeco with another Vertex drug (VX-809), that work in combination to target the DF08 mutations. They are also testing another compound, VX-661.

 

[stranger]

Eight of 112 demonstrated some level of what was generally accepted efficacy in the DDF508 trial of Kalydeco. Question: if they applied the same standards in the DDF508 809 + Kalydeco Ph3 going on right now... would that number been higher than eight? Less than eight? Same? Has anyone asked that question before? Paging Dr. Flume, paging Dr. Flume.