PTC 124-2b Clinical Trial

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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

Discussions

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

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Comment by Barb Rush on December 13, 2008 at 6:21pm
Erica, Thanks, I have tried milk, Choclate milk, water, milk & water. I think it is more of a mind game. I really don't know!!!! I am going to try prizes frot eh next couple of time he takes it and see if that works. He wasn't able to go see Santa today until he drank it & mom my left with my little and boy did he drink it down.
Comment by Erica on December 13, 2008 at 5:23pm
Hi Barb,
Are you mixing the powder with water or with milk? My son only takes it with milk. He complained about the taste at the very beginning (one or two days), and since that we haven't had any problems.
Comment by Barb Rush on December 12, 2008 at 12:23pm
Hi all I am alittle late joining the group, but my son Brenden just started the trial on Dec 4th. and it has been a rough go. I can not get him to take the meds for nothing. I've tried stickers, a new toy car for so many stickers, give money, it will help you get stronger. Nothing seems to work. any idea.

About the biopsy. we never told him exactly what it was I just called it a procedure.that way it wouldn't toally freak him out. Hed had it doen on Nov.20th and like I said we started the Drug??? on the 4th of Dec.
Comment by Erica on December 5, 2008 at 8:16am
Hello Brad,
Before starting the trial, Matias knew that there was “a problem with his muscles”.
We told him that we had found a great team of doctors and we were convinced that they could help us. We stressed that we are a TEAM (mom and dad, doctors, family and friends, teachers, our bosses… and of course himself) and that we should work all together. This treatment would require a big effort for all and we were all willing to make it, but he had to be too. We explained the role of each team member (for example, his teachers would help him not get behind because of the classes that he was going to miss, etc.) and his part, which is the most important. And we assured him, that we would do everything we could to make it easier for him (e.g. see if we could try using a local anesthetic for the blood work so that it wouldn’t hurt, etc.).
It was a long talk… at the end of which he said that he wanted to do it.
Comment by brad stringer on November 26, 2008 at 1:09am
Erica,
Just wondering how you prepared Mati just before starting the whole clinical trial and pre-screening process? What did you tell him about the upcoming test he would be doing?
Comment by Erica on November 17, 2008 at 12:21pm
For us, the deciding factor was “time”. As I mentioned, Mati is already 8 years old. He was diagnosed when he was 3, and we have seen the (thank God slow) progression of the disease.
We thought that the sooner we start the treatment, the better.
We had already faced the decision whether to participate in the 2a phase, but then decided not to, because it was not therapeutic. So we had been processing the whole idea for quite some time.

To help him with the blood work, we reasoned with him. Mati is old enough to understand that sometimes you have to do things you don’t like. Then, the first time he acted really nicely, we gave him a playstation game he wanted, to show him that we were happy and proud. But we don’t give him presents every time, because we don’t want to send a wrong message: the present should be a consequence of his nice behavior, and not the cause.
Of course, with younger boys it’s different!
For taking the medication, as he didn’t like the taste at first, we gave him a gummy bear along with it to disguise the taste (the study coordinator was ok with that). Now, he got used to the taste, but he still gets one gummy bear each time and he looks forward to it (he loves them).
Comment by brad stringer on November 16, 2008 at 5:35pm
Paul..

Brilliant!! Its obvious that you are a caring, intuitive, great dad! Too many parents shower their kids with big expensive items, not giving any thought to what would be most valuable to their child.
My little guy Skyler has a new collection of bottle caps- he loves collecting them....so I am always on the lookout.
Whenever I get them a new plastic toy, the charm has worn off minutes later, never to be played with again.
I rarely buy new plastic anything.
He also loves making potions with various fluids...messy but fun...
Comment by Paul Johnson on November 16, 2008 at 12:44pm
The present was just a $5.00 stuffed "Hop-On-Pop" that we got a Kohl's. We knew he'd like it (because he kept 'stealing' his cousins when we were on vacation together). So it doesn't need to be something extravagant... just something you know they'll like.
Comment by brad stringer on November 16, 2008 at 12:19am
Paul...
The present idea, and your input about how you did it is a great idea...as well as the music player.Thanks for these thoughts....they are very helpful to us all.
So...what was in the present, and did he like it?
Comment by Paul Johnson on November 15, 2008 at 11:27pm
Hey Angela...
Yes we were the on Weds, and Lyndsay was with us. We got there about 8:30 AM and were finished by about 2:30. I know another family was coming in, but I thought I heard her mention to someone they were coming from Upstate NY. We'll have to coordinate in case in the future we are there on the same day and we can meet and grab lunch or such... Good luck with everything...
 

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