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PTC124 for Nonsense Mutations
- Thread starter kitomd21
- Start date
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Ratatosk</b></i>
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Ratatosk</b></i>
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Ratatosk</b></i>
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote></div>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Ratatosk</b></i>
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote>
I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Ratatosk</b></i>
<br />
<br />Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote>
<br />
<br />I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<br />
<br />
<br />
<br />Wouldn't it eliminate the nonsense mutation and in essense make the indvidual a carrier of the delta f508 gene? Just a thought...</end quote>
<br />
<br />I was thinking along these lines too but I guess it just seemed to simple <img src="i/expressions/face-icon-small-smile.gif" border="0"> Thanks for the thought! Here is hoping it will help<img src="i/expressions/beer.gif" border="0">
<br />
<br />
I'm still trying to find a list of mutations considered as "nonsense mutations". I believe 80% of all CF cases are due to dd508 (can't remember where I heard that...) so it would seem that the 10% mentioned in this article as "nonsense" may apply to many others that have alternate mutations, thus the drug may help. As noted, it's in phase 2 trials.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
I'm still trying to find a list of mutations considered as "nonsense mutations". I believe 80% of all CF cases are due to dd508 (can't remember where I heard that...) so it would seem that the 10% mentioned in this article as "nonsense" may apply to many others that have alternate mutations, thus the drug may help. As noted, it's in phase 2 trials.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
I'm still trying to find a list of mutations considered as "nonsense mutations". I believe 80% of all CF cases are due to dd508 (can't remember where I heard that...) so it would seem that the 10% mentioned in this article as "nonsense" may apply to many others that have alternate mutations, thus the drug may help. As noted, it's in phase 2 trials.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
I'm still trying to find a list of mutations considered as "nonsense mutations". I believe 80% of all CF cases are due to dd508 (can't remember where I heard that...) so it would seem that the 10% mentioned in this article as "nonsense" may apply to many others that have alternate mutations, thus the drug may help. As noted, it's in phase 2 trials.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
Here is an excerpt...
A No-Nonsense Cure for Cystic Fibrosis
Wednesday July 23, 2008
PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
I'm still trying to find a list of mutations considered as "nonsense mutations". I believe 80% of all CF cases are due to dd508 (can't remember where I heard that...) so it would seem that the 10% mentioned in this article as "nonsense" may apply to many others that have alternate mutations, thus the drug may help. As noted, it's in phase 2 trials.
<br />
<br />Here is an excerpt...
<br />
<br />A No-Nonsense Cure for Cystic Fibrosis
<br />Wednesday July 23, 2008
<br />PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
<br />
<br />PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
<br />
<br />Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
<br />
<br />Here is an excerpt...
<br />
<br />A No-Nonsense Cure for Cystic Fibrosis
<br />Wednesday July 23, 2008
<br />PTC Therapeutics, the pharmaceutical company that is in phase 2 testing of a promising new drug called PTC124, got a shot in the arm last week when Genzyme Corporation bought in to a deal to partner with PTC in marketing the drug once it receives FDA approval.
<br />
<br />PTC124 targets a group of mutations known as nonsense mutations, in which a piece of code that appears in a gene stops the gene from performing its function. It is one of several drugs being studied that researchers hope will be able to do something that has yet to be accomplished by actually fixing the problem at its root rather than the current treatments that merely control symptoms and slow progression of the disease.
<br />
<br />Nonsense mutations are responsible for about 10% of cystic fibrosis cases, and 13% of all cases of another devastating genetic disease, Duchenne muscular dystrophy. This may seem like a small number of people that stand to benefit from PTC124, but for those with nonsense mutations the potential is huge. For now, the only way to access the drug is by participating in the clinical trial but if all goes well the drug may be available to the general public in 2011.
List of nonsense mutations...
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
List of nonsense mutations...
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
List of nonsense mutations...
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
List of nonsense mutations...
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
Dear Mrs. Schwartz,
Thank you for your reply.
Please find below a list of the known nonsense mutations in CF:
E60X
Q890X
R1162X
W1282X
Y1092X
R75X
3849+10kbC>T
W1089X
S1196X
G542X
G330X
R553X
Q493X
Y122X
R1158X
S1225X
K710X
W846X
I hope this information is useful. Please feel free to contact us again should you have additional questions.
Sincerely,
Alissa M. Coslit
Patient and Professional Advocacy
PTC Therapeutics
100 Corporate Court
South Plainfield, NJ 07080
Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
Fax: 908 222-7231
acoslit@ptcbio.com
List of nonsense mutations...
<br />
<br />Dear Mrs. Schwartz,
<br />
<br />Thank you for your reply.
<br />
<br />Please find below a list of the known nonsense mutations in CF:
<br />
<br />E60X
<br />Q890X
<br />R1162X
<br />W1282X
<br />Y1092X
<br />R75X
<br />3849+10kbC>T
<br />W1089X
<br />S1196X
<br />G542X
<br />G330X
<br />R553X
<br />Q493X
<br />Y122X
<br />R1158X
<br />S1225X
<br />K710X
<br />W846X
<br />
<br />I hope this information is useful. Please feel free to contact us again should you have additional questions.
<br />
<br />Sincerely,
<br />
<br />Alissa M. Coslit
<br />Patient and Professional Advocacy
<br />PTC Therapeutics
<br />100 Corporate Court
<br />South Plainfield, NJ 07080
<br />Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
<br />Fax: 908 222-7231
<br />acoslit@ptcbio.com
<br />
<br />Dear Mrs. Schwartz,
<br />
<br />Thank you for your reply.
<br />
<br />Please find below a list of the known nonsense mutations in CF:
<br />
<br />E60X
<br />Q890X
<br />R1162X
<br />W1282X
<br />Y1092X
<br />R75X
<br />3849+10kbC>T
<br />W1089X
<br />S1196X
<br />G542X
<br />G330X
<br />R553X
<br />Q493X
<br />Y122X
<br />R1158X
<br />S1225X
<br />K710X
<br />W846X
<br />
<br />I hope this information is useful. Please feel free to contact us again should you have additional questions.
<br />
<br />Sincerely,
<br />
<br />Alissa M. Coslit
<br />Patient and Professional Advocacy
<br />PTC Therapeutics
<br />100 Corporate Court
<br />South Plainfield, NJ 07080
<br />Office <img src="i/expressions/face-icon-small-sad.gif" border="0">908)912-9221
<br />Fax: 908 222-7231
<br />acoslit@ptcbio.com