PTC 124-2b Clinical Trial


PTC 124-2b Clinical Trial

Various issues re: PTC 124 Clinical Trials for current or prospective participants worldwide

Members: 31
Latest Activity: May 1, 2018


PTC124 now has a name...

Started by Paul Johnson. Last reply by Paul Johnson Feb 3, 2009. 1 Reply

PTC124 in the courts

Started by Paul Johnson. Last reply by Paul Johnson Dec 22, 2008. 4 Replies

Is the 2b Trial Full?

Started by Paul Johnson. Last reply by brad stringer Dec 4, 2008. 1 Reply

Comment Wall


You need to be a member of PTC 124-2b Clinical Trial to add comments!

Comment by neeraj on June 1, 2009 at 3:07am

Thanks for your message and info.Good luck with a new start.

Comment by neeraj on April 22, 2009 at 1:18am
Anyone in the 2b extension trial,Please share weather any additional biopsies are required for this part of the trial or not.My son will finish the 2b trial in october and will start the ext trial then.Any other thoughts are very welcome and will be very helpful.


Comment by neeraj on April 1, 2009 at 12:49pm
Hi Erica,

Thankyou for your input and sincerely hope Mati does extremely well and this truly offers a life long therapy for a full almost normal life to you son.We still have few months until october to go through the 2b trial and we really are waiting towards the end of this 2b and start of the extension study.Good Luck.

Comment by Erica on April 1, 2009 at 8:18am
Hi Neeraj,
In our case, the last visit of the 2b phase was, at the same time, the first visit of the extension study, so we didn't have to wait at all.
Take care!
Comment by neeraj on March 29, 2009 at 11:36am
Hi Everyone,
My son has been in the 2b trial for now over 5 months.The question I have is would he start the 2b extension straight away or would there be some time gap before he starts the extension trial.Please share your experience.

Aran's Dad
Comment by Erica on March 27, 2009 at 8:43am
Hello everyone,
I just wanted to share with you that Mati has started the 2b extension study this week.
I am really excited!
Comment by Cathie Bullis on February 28, 2009 at 1:03pm
We are having more luck giving Michael the medicine with chocolate mint flavored pudding. Perhaps this will help others having trouble.
We are seeing no improvement since starting the study.
Comment by Cathie Bullis on February 25, 2009 at 11:56pm
I am sorry about the study, we know how very difficult it is too take that medication, when you already have a sick child. I am not sure Michael can keep it up and some days don't work as well as others.

Good Luck for all of you.
Comment by Barb Rush on February 25, 2009 at 11:17pm
Cathie, We had a heck of a time, nothing worked. So long story short we are no longer in the trial. We tried everying to, pudding, juice chaser, you name it. Nothing... Oh well it is just a trial right. but my son was one of the younger boys. he is just 5 1/2
Comment by Cathie Bullis on February 25, 2009 at 10:47pm
I hope all the children in the PTC 124 are doing well. Our grandson is in the trial and is having a heck of the time getting the medicine down. The milk, juice, pudding, water, etc. are not so successful. Anyone having any better luck with making this an easier process?

We will know more about the agility test in a couple of weeks (every 6 weeks).

Cathie Bullis

Members (31)


Need help using this community site? Visit Ning's Help Page.



© 2022   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service