PTC124

Z

zoeg

New member
Hello!

I was curious if any of you, or your children are trying the phase II drug PTC124? It sure sounds promising for those with a nonsense mutation and I can't find anything online about how the actual trial is going. My daugter has the delta f508, but she also has Y1092x which is so rare, but is a nonsense. Anyway, if anyone has any info on this I would love to hear what you have to say.

Thanks,
Zoe mom to Kate wCF
 
Z

zoeg

New member
Hello!

I was curious if any of you, or your children are trying the phase II drug PTC124? It sure sounds promising for those with a nonsense mutation and I can't find anything online about how the actual trial is going. My daugter has the delta f508, but she also has Y1092x which is so rare, but is a nonsense. Anyway, if anyone has any info on this I would love to hear what you have to say.

Thanks,
Zoe mom to Kate wCF
 
Z

zoeg

New member
Hello!

I was curious if any of you, or your children are trying the phase II drug PTC124? It sure sounds promising for those with a nonsense mutation and I can't find anything online about how the actual trial is going. My daugter has the delta f508, but she also has Y1092x which is so rare, but is a nonsense. Anyway, if anyone has any info on this I would love to hear what you have to say.

Thanks,
Zoe mom to Kate wCF
 
M

Mommafirst

Guest
We are not on the trial, but I'm curious too!!! My daughter has the W1282X nonsense mutation -- I've been keeping my eyes peeled on this one!!
 
M

Mommafirst

Guest
We are not on the trial, but I'm curious too!!! My daughter has the W1282X nonsense mutation -- I've been keeping my eyes peeled on this one!!
 
M

Mommafirst

Guest
We are not on the trial, but I'm curious too!!! My daughter has the W1282X nonsense mutation -- I've been keeping my eyes peeled on this one!!
 
M

mom2leila

New member
Hi Zoe,

We are watching the trials closely too. In the current Phase II study you must be at least 12 years to participate. Another Phase II study is being planned to include children as young as 6 years. PTC 124 is made by PTC therapeutics. You can read more about it on their website, www.ptcbio.com . On the website is questions and answers about PTC 124 with lots in info. plus a link to the clinical trial website. Somewhere on their site you can sign up to get updates sent from PTC therapeutics. I get these from time to time. PTC 124 is also being tested in Duchenne's Muscular Dystrophy, since that is also caused by a stop mutation. Interesting stuff. We are hopeful!
 
M

mom2leila

New member
Hi Zoe,

We are watching the trials closely too. In the current Phase II study you must be at least 12 years to participate. Another Phase II study is being planned to include children as young as 6 years. PTC 124 is made by PTC therapeutics. You can read more about it on their website, www.ptcbio.com . On the website is questions and answers about PTC 124 with lots in info. plus a link to the clinical trial website. Somewhere on their site you can sign up to get updates sent from PTC therapeutics. I get these from time to time. PTC 124 is also being tested in Duchenne's Muscular Dystrophy, since that is also caused by a stop mutation. Interesting stuff. We are hopeful!
 
M

mom2leila

New member
Hi Zoe,

We are watching the trials closely too. In the current Phase II study you must be at least 12 years to participate. Another Phase II study is being planned to include children as young as 6 years. PTC 124 is made by PTC therapeutics. You can read more about it on their website, www.ptcbio.com . On the website is questions and answers about PTC 124 with lots in info. plus a link to the clinical trial website. Somewhere on their site you can sign up to get updates sent from PTC therapeutics. I get these from time to time. PTC 124 is also being tested in Duchenne's Muscular Dystrophy, since that is also caused by a stop mutation. Interesting stuff. We are hopeful!
 
R

rarab43

Guest
My son who is 21 was in the first phase. The last time he was at clinic they told him they were going to a big meeting and would be contacting him about enrolling again. I need to contact them again I guess. He finished the first phase in Dec of 05. In fact he was the first one in the US to take the medicine. There was definitely a change in his results from before he took the med and after- they did a nasal test to monitor the results. Our researcher described it as his numbers after the med was not normal but less like CF than before. We were enrolled in Birmingham Al. Susan
 
R

rarab43

Guest
My son who is 21 was in the first phase. The last time he was at clinic they told him they were going to a big meeting and would be contacting him about enrolling again. I need to contact them again I guess. He finished the first phase in Dec of 05. In fact he was the first one in the US to take the medicine. There was definitely a change in his results from before he took the med and after- they did a nasal test to monitor the results. Our researcher described it as his numbers after the med was not normal but less like CF than before. We were enrolled in Birmingham Al. Susan
 
R

rarab43

Guest
My son who is 21 was in the first phase. The last time he was at clinic they told him they were going to a big meeting and would be contacting him about enrolling again. I need to contact them again I guess. He finished the first phase in Dec of 05. In fact he was the first one in the US to take the medicine. There was definitely a change in his results from before he took the med and after- they did a nasal test to monitor the results. Our researcher described it as his numbers after the med was not normal but less like CF than before. We were enrolled in Birmingham Al. Susan
 
A

AnD

New member
Huh. I guess that means that there is a good reason for knowing what 2 genes I have! I wonder if my doctor's <i>do</i> know my mutations... and hello from Birmingham <img src="i/expressions/face-icon-small-wink.gif" border="0"> .
 
A

AnD

New member
Huh. I guess that means that there is a good reason for knowing what 2 genes I have! I wonder if my doctor's <i>do</i> know my mutations... and hello from Birmingham <img src="i/expressions/face-icon-small-wink.gif" border="0"> .
 
A

AnD

New member
Huh. I guess that means that there is a good reason for knowing what 2 genes I have! I wonder if my doctor's <i>do</i> know my mutations... and hello from Birmingham <img src="i/expressions/face-icon-small-wink.gif" border="0"> .
 
R

rarab43

Guest
Hi from Tuscaloosa- do you go to UAB? We just switched there from Childrens. Susan
 
R

rarab43

Guest
Hi from Tuscaloosa- do you go to UAB? We just switched there from Childrens. Susan
 
R

rarab43

Guest
Hi from Tuscaloosa- do you go to UAB? We just switched there from Childrens. Susan
 
H

hopefullmom

New member
I am also keeping up with everything that is going on with the PTC124 study, my daughter is 4 and will turn 5 in July, we go to the clinic in Houston, but I keep in contact with all the researchers in Alabama, theywill be going to a conference within the next week, and if at all possible Maci will get to be in the study. YEA!! She has W1282X and DF508. I believe they have already started on children in Europe. Let me know if you all know anything else. Thanks
 
H

hopefullmom

New member
I am also keeping up with everything that is going on with the PTC124 study, my daughter is 4 and will turn 5 in July, we go to the clinic in Houston, but I keep in contact with all the researchers in Alabama, theywill be going to a conference within the next week, and if at all possible Maci will get to be in the study. YEA!! She has W1282X and DF508. I believe they have already started on children in Europe. Let me know if you all know anything else. Thanks
 
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