Proteostasis: F508del Research

[GenH]

Proteostasis have identified multiple compounds that improve the folding and trafficking of F508del. These compounds have a similar level of activity to VX809, and when added to VX809, about 40% of normal function is reached. Trials are planned to start in 2015.

I've added more info and some images here: http://sixtyfiverosesblog.wordpress.com/2013/08/07/proteostasis-f508del-research/

 

[rosesixtyfive]

Thanks for posting GenH. Do you get the feeling we are on the edge of a new frontier? rosesixtyfive, mother of Sam, 3 years old, ddf508

 

[GenH]

Definitely, there are many companies working on CFTR medications now- I've come across all of these companies and there are probably more:

• 3. Calista Therapeutics – Procaftor
• 3. Galapagos – GLPG1
• 3. Genzyme – CFTR Research
• 3. N30 – N6022 & N91115
• 3. Pfizer – F508del Research
• 3. ProQR – RNA Correction
• 3. Proteostasis – F508del Research
• 3. PTC – Ataluren
• 3. Vertex – Kalydeco
• 3. Vertex – Second Generation Correctors
• 3. Vertex – VX809 & VX661 & VX983