Experimental Hadassah-inspired pill is 'promising' in repairing cystic fibrosis gene

Allie

New member
Stop or X mutations are a class 1, Df508 is a class 2, and I think it's a delection.....PTC124 won't do anything for it.
 

Allie

New member
Stop or X mutations are a class 1, Df508 is a class 2, and I think it's a delection.....PTC124 won't do anything for it.
 

Allie

New member
Stop or X mutations are a class 1, Df508 is a class 2, and I think it's a delection.....PTC124 won't do anything for it.
 

lilmac7

New member
ok....so I am delta f508 and R355X, would they do anything for me? Would that mean then that the PTC124 would correct the defective R355X but do nothing for the f508 and therefore still causing correction of the CF symptoms, kinda like someone being a carrier and having no symptoms? Or does that mean that once a cfer has a stop mution in the mix then the drug cant be of any benefit?
 

lilmac7

New member
ok....so I am delta f508 and R355X, would they do anything for me? Would that mean then that the PTC124 would correct the defective R355X but do nothing for the f508 and therefore still causing correction of the CF symptoms, kinda like someone being a carrier and having no symptoms? Or does that mean that once a cfer has a stop mution in the mix then the drug cant be of any benefit?
 

lilmac7

New member
ok....so I am delta f508 and R355X, would they do anything for me? Would that mean then that the PTC124 would correct the defective R355X but do nothing for the f508 and therefore still causing correction of the CF symptoms, kinda like someone being a carrier and having no symptoms? Or does that mean that once a cfer has a stop mution in the mix then the drug cant be of any benefit?
 

anonymous

New member
My son who was in the study last yr only has one stop mutation so Im sure it would work. I guess it would in essence make you a carrier. We were told that his other mutation would be very hard to do anything about. His mutations are E60X and 621+1G->T. Susan
 

anonymous

New member
My son who was in the study last yr only has one stop mutation so Im sure it would work. I guess it would in essence make you a carrier. We were told that his other mutation would be very hard to do anything about. His mutations are E60X and 621+1G->T. Susan
 

anonymous

New member
My son who was in the study last yr only has one stop mutation so Im sure it would work. I guess it would in essence make you a carrier. We were told that his other mutation would be very hard to do anything about. His mutations are E60X and 621+1G->T. Susan
 
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2perfectboys

Guest
for the poster that had the son in the study, did u notice any difference in symptoms?
 
2

2perfectboys

Guest
for the poster that had the son in the study, did u notice any difference in symptoms?
 
2

2perfectboys

Guest
for the poster that had the son in the study, did u notice any difference in symptoms?
 
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