Anything for Delta f508

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Anything for Delta f508

Postby vicky257 » Wed 11 Jan, 2012 13:43

I have read al lot lately about trials for other mutations and just wondering if there is anything going on DF508, my daughter has double and knowing it is the most common thought it would be one of the most looked at? x
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Postby Maddie's Family » Wed 11 Jan, 2012 13:55

Vertex have been trialling a combination small molecular therapy for DeltaF508 using both VX-770 (KALYDECO) and VX-809. Part 1 of the Phase 2 study has been completed with no major safety issues and some results which range between equivocal and potentially promising. Patients are being enrolled in the USA for Part 2 of the Phase 2 study.
More details can be found here.

And do not forget that it is hoped that the full Phase 2 multi-dose trial for the proposed gene therapy treatment will start at some point this year. Unlike the small molecule treatments being explored by Vertex, it is hoped that the gene therapy will be effective regardless of which mutation(s) a patient carries.
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Postby Arginine Avenger » Wed 11 Jan, 2012 14:42

As said above, there are drugs on development for DF508, but drug development unfortunately doesn't just go by what is most common. They need to work on the genes that are "easiest" to crack with various techniques. Then with each drug they learn more and more, which then can be applied to other genes. I'm not a doctor but I assume DF 508 is the toughest nut to crack so it needs to be something they work towards progressively. An absolutely successful drug for DF 508 will of course be the holy grail but we just need to hang in there for a while!

Luminous beings are we, not this crude matter.
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