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

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Comment by Perlita & Gordy Hains on December 16, 2009 at 10:50pm
thank you all so very much for responding. My family has high hopes for Ataluren and possibly utrophin & myostatn. Vonda, I think we live close so lets keep in touch - would luv to meet sometime - happy holidays to everyone!
Comment by Vonda Fox on December 14, 2009 at 4:26pm
Let's keep in touch over the next weeks/months. Hopefully we will have good news to report!!!
Comment by Donna on December 14, 2009 at 4:12pm
Our son, Kyle (6 1/2 yrs old), just started the final phase I guess I'll call it last week. I am guessing that he was either on a placebo or a low dose as we did not notice much change, BUT thankfully there was not much to notice anyway as he is doing really well. Now that he is on the high dose, I will be interested to see if his balance seems any better on stairs - that is really the most noticeable thing with him as he runs around like crazy most days. Our nurse told us that we might hear in about six months what he was taking during this last phase. I did inquire as to why they wouldn't be doing another muscle biopsy during this final phase and was told that well, they already know what the muscle change was/is because some of the kids were on the high dose, some on the low dose and some on placebo. So, that made sense. I too am hopeful that this will be a long term benefit to our boys.
Comment by Vonda Fox on December 14, 2009 at 3:20pm
Our son, Ryan (13 yrs. old), just completed the 48-week double blind study on Dec.10th. We are guessing he was on the placebo, because we didn't see any noticeable improvement. He was one of the last boys to finish that phase of the study (164 boys total). Now they plan to get all the data together, study it, and compare the different groups (those who were on the drug and those who weren't , the older boys compared to the younger ones, those who were taking steroids and those who weren't). They are hoping to get some results out possibly by spring. We don't know how long it will take for it to actually be approved, maybe 6 months to a year. In the meantime, we are excited that Ryan is on the drug for sure! We are hoping & praying that ataluren will be a success!
Comment by Perlita & Gordy Hains on December 13, 2009 at 9:55pm
can anyone update me on PTC trials? Our Levi is 3 and has stop codon. When I met Lee Sweeney at conference he said as soon as approved to get Levi on asap - can anyone shed light on timeline? thx
Comment by Perlita & Gordy Hains on July 1, 2009 at 10:00pm
MY son Levi is a patient of Dr. Finkel at CHOP. He is almost 3. We learned that he has point mutation at exon 23. We have high hopes for Ataluren. Lets keep the hopes alive.
Comment by Cathie Bullis on June 2, 2009 at 5:47pm
Michael has his 6 week visit again tomorrow morning, so we will see if there is anymore news.
Cathie Bullis
Comment by Sara Savalli on June 2, 2009 at 4:45pm
no i do not think they tell us
Comment by Donna on June 1, 2009 at 3:15pm
For those of you in the extension phase, have you found out whether or not your son had the drug or had the placebo?
Comment by Sara Savalli on June 1, 2009 at 3:10pm
we are now in the next phase of the trial as of end of april, and no more biopsies, other than the 2 we already did. blood work, but no biopsy.

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