K
Kaethe108
Guest
Is there nothing new about Ataluren?
Why is nothig happen with that?
Why is nothig happen with that?
PTC124 (Ataluren)
- Thread starter missT
- Start date
Nothing has happened since 6/8/12. They have continued studies on muscular distophy. If you look at the CF drug pipeline it is still there...but I want to say I heard it wasn't going anywhere....I could be totally wrong and I hope maybe Genevieve will jump in for you on this one. My memory fails me 
I was watching this one to sense Abby has a stop mutation.
I was watching this one to sense Abby has a stop mutation.
K
Kaethe108
Guest
So I see that your daughter has one F508del and one stop mutation (class 1)?
thats what our daughter has.
And how were you able to get Kalydeco? With these mutations?
what are the benefits that you are experiencing since she takes Kalydeco?
i would be happy to hear your story!
thats what our daughter has.
And how were you able to get Kalydeco? With these mutations?
what are the benefits that you are experiencing since she takes Kalydeco?
i would be happy to hear your story!
Nothing has happened since 6/8/12. They have continued studies on muscular distophy. If you look at the CF drug pipeline it is still there...but I want to say I heard it wasn't going anywhere....I could be totally wrong and I hope maybe Genevieve will jump in for you on this one. My memory fails me
Sorry but I have not heard anything either since the middle of last year. I heard that more work was needed with Ataluren and also that there were further meds being developed, but I have not read anything official. I also noticed that they are continuing studies with muscular dystrophy (according to clinicaltrials.gov).
http://ptct.client.shareholder.com/releasedetail.cfm?ReleaseID=746125 Just so happens that I got this email today and well I'm not sure it really says all that much but you can read it. It acts like they are continuing.
K
Kaethe108
Guest
Hmm, thanks for the info!
I have the feeling that they ran out of money. Suddenly we heard nothing anymore, though the results from the trials were not too bad. Also the patient's service did not answer emails anymore, which they always did before.
So its good news that there will be money running!!
I have the feeling that they ran out of money. Suddenly we heard nothing anymore, though the results from the trials were not too bad. Also the patient's service did not answer emails anymore, which they always did before.
So its good news that there will be money running!!
CFF has said they didn't get the results they needed from the phase 3 trials of ataluren for cf so they are trying to figure out what to do next with it. (Higher doses, pairing it with another drug, etc.) This was discussed at the national volunteer conference earlier this month. Very disappointing since both my son's mutations are nonsense.