PTC124 Phase 2a Extension Study Families

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PTC124 Phase 2a Extension Study Families

This group is intended to be for families of the 38 boys who participated in the clinical trial of PTC124 phase 2a, to be able to share with each other the progress of our boys while on this extension study.

Members: 24
Latest Activity: Jan 19, 2016

Discussions

New consents

Started by Angela Feb 3, 2009. 0 Replies

Being Bad or Self testing

Started by Angela Oct 16, 2008. 0 Replies

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Comment by Trinh Nguyen on October 16, 2013 at 9:37pm

My son is just in the trial phase III today.

Comment by Chad Studebaker on September 2, 2011 at 6:31am
Comment by Gretchen on January 17, 2010 at 8:08pm
Wow, no one's posted here in forever. Anybody mix their meds with soy milk?
Comment by Angela on January 30, 2009 at 12:15pm
The MRI portion is optional, but will hopefully help remove biopsies from future trials, so we do that part "for the greater good".
They never give results of the clinical trials, because it can affect the outcome of the trial. We do not keep a journal (for the trial).
We were promised results from the 2a trial, but 3 years later and we haven't recieved them and I don't believe we ever will.
The 2a extension patients are getting the medication and know the dosage.
I hope this answers some of your questions!
Ang :)
Comment by Michelle on January 30, 2009 at 9:47am
I've been following some of this online. This 2a extension apparently requires biopsies and MRI's, but they don't share the results with you? Is it because you're keeping a journal and they don't want to bias you? Will they tell you at the very end? If not, did they explain why? Do you know that you're definitely getting the drug and/or the dosage? My understanding is that the 2a extn isn't a blind trial like the phase 2b one. Sorry for all the questions!
Comment by Angela on January 30, 2009 at 12:55am
Today should officially be Jack's 6 month biopsy (we pushed it back to next week) and I can't help but reflecting on the last six months. It really has been a rollercoaster ride and surprisingly more stressful and exhausting than we expected. Is it the length of time, the travel, the expectation, etc. that make this seem so much more then the original trial? We are still very hopeful!! I just wish that we had some information to know what is going on in his little body . . . Oh, what I would give for those biopsy results!!!!!
What I keep thinking is Jack should have his 5th and LAST biopsy next week!!! We can live without doing that ever again.
I hope you all and your boys are holding up well and seeing only the good in this whole "trial".
Ang
Comment by Joanne Wechlser on December 17, 2008 at 10:55am
Hi there, I noticed, not much discussion here these days. Feel free to check out our blog at www.caringbridge.org/visit/adamwechsler
Comment by Jill Castle on October 16, 2008 at 9:39am
Hang in there gals! It's all worth it. We have been on the drug now for 9 weeks and there has been some pretty big changes. I know that some of the boys haven't seen the dramatic results Anthony has and others have but we need to remember even if this just stops the progression it's going to be a downright miracle and worth all the pain.
www.caringbridge.com/visit/anthony
Comment by Joanne Wechlser on September 29, 2008 at 5:06pm
Hi Ellen thanks for posting. We are just getting started. I started a caring bridge for Adam as well, if anyone would like to follow our progress. www.caringbridge.org/visit/adamwechsler
Comment by Ellen Wagner on September 28, 2008 at 4:21pm
We are back from Cinn and Tim is on the PTC. Hurrah!!! What a roller coaster ride the last two weeks have been. All of the trauma of the trial and travel combined with the excitement of starting the PTC. All in all the process has gone smoothly. The arm biopsy was a little more traumatic during surgery but much easier healing than the foot was. Tim did not even take a tylenol. The blood draws are also difficult but short lived trauma. This last trip to Cinn the vein blew in Tim's arm as they were putting in the IV. They had to put it in Tim's hand instead and that was a little more difficult for mobility. Tim also wasn't bleeding very well because of the position of the IV so they had to use a heating pad all day on that hand. Tim did get through it ok. Tim says the PTC tastes disgusting and it does make him gag. We bought a battery powered milk frother and that works great to mix the powder so Tim doesn't see it as he drinks. Jack Knight said Tim will get used to the taste. He doesn't even notice now. We are hoping that happens soon. We are looking forward to a few weeks of being home and getting back into a routine. This travel has been hard on everyone. Tim does not even like to travel for vacation. The travel has also been hard on our other son. Chris is a junior in high school. He is too old to miss school and come with us but not old enough to stay home by himself. I think the next trip I will take Tim myself and my husband will stay home with Chris and the dogs. Good luck to the others who are about to start. If anyone has any more specific questions let me know.
Gretchen, we would love to get together. Let's talk about a date.

Ellen
 

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