PTC124

anonymous

New member
Hi i was just wondering if anyone was involved in this clinical trial. thanks


Melissa mom to dylan 7 no cf and caleb 4wcf
 

anonymous

New member
My son Will who is 20 just got through with the study. He was enrolled in Birmingham Alabama and was the first one enrolled in the US. He has to go back next week for his final checkup. There is one more person enrolled in Birmingham and I think the other sites are still trying to get up and started. The medicine did show a change in his nasal PD- a nose sweat test is how the dr said was the best way to describe it- it was not normal but less CF than his baseline one. The higher dose did not show any more change than the lower one. I also heard them say they were getting good results in the study in Israel- 99% of the Jewish population has this mutation. We will have to wait for the next phase after they get all the other people done. The only side effects Will had was some burning in his throat right after he took the med-lasted a minute and burning when he went to the bathroom the first time in the morning. Susan
 
Top