MargaritaChic
New member
Does anyone have any information or links regarding PTC 124?
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PTC 124 ?
- Thread starter MargaritaChic
- Start date
MargaritaChic
New member
Does anyone have any information or links regarding PTC 124?
MargaritaChic
New member
Does anyone have any information or links regarding PTC 124?
MargaritaChic
New member
Does anyone have any information or links regarding PTC 124?
MargaritaChic
New member
Does anyone have any information or links regarding PTC 124?
M
Mommafirst
Guest
I don't have a lot of information -- but you might try running a search on the main forms page ofthis sitr -- as it has been discussed before. I know that PTC 124 is now in phase III trials for adults over 18. Thus far it has shown great promise for any CFer who has at least one mutation with an X at the end. I don't know if/when it will go to trial for younger patients. If you google it you will find that it is also showing promise for helping duchene musular dsytrophy. I believe it helps to fix the "stop" in the genetic coding and thus gets the body to read through and create the protein that was not working. So in CF it with get the CFTR to work properly and the idea would be that with the drug (daily) a patient would be more like a carrier.
I hope this helps.
I hope this helps.
M
Mommafirst
Guest
I don't have a lot of information -- but you might try running a search on the main forms page ofthis sitr -- as it has been discussed before. I know that PTC 124 is now in phase III trials for adults over 18. Thus far it has shown great promise for any CFer who has at least one mutation with an X at the end. I don't know if/when it will go to trial for younger patients. If you google it you will find that it is also showing promise for helping duchene musular dsytrophy. I believe it helps to fix the "stop" in the genetic coding and thus gets the body to read through and create the protein that was not working. So in CF it with get the CFTR to work properly and the idea would be that with the drug (daily) a patient would be more like a carrier.
I hope this helps.
I hope this helps.
M
Mommafirst
Guest
I don't have a lot of information -- but you might try running a search on the main forms page ofthis sitr -- as it has been discussed before. I know that PTC 124 is now in phase III trials for adults over 18. Thus far it has shown great promise for any CFer who has at least one mutation with an X at the end. I don't know if/when it will go to trial for younger patients. If you google it you will find that it is also showing promise for helping duchene musular dsytrophy. I believe it helps to fix the "stop" in the genetic coding and thus gets the body to read through and create the protein that was not working. So in CF it with get the CFTR to work properly and the idea would be that with the drug (daily) a patient would be more like a carrier.
I hope this helps.
I hope this helps.
M
Mommafirst
Guest
I don't have a lot of information -- but you might try running a search on the main forms page ofthis sitr -- as it has been discussed before. I know that PTC 124 is now in phase III trials for adults over 18. Thus far it has shown great promise for any CFer who has at least one mutation with an X at the end. I don't know if/when it will go to trial for younger patients. If you google it you will find that it is also showing promise for helping duchene musular dsytrophy. I believe it helps to fix the "stop" in the genetic coding and thus gets the body to read through and create the protein that was not working. So in CF it with get the CFTR to work properly and the idea would be that with the drug (daily) a patient would be more like a carrier.
I hope this helps.
I hope this helps.
M
Mommafirst
Guest
I don't have a lot of information -- but you might try running a search on the main forms page ofthis sitr -- as it has been discussed before. I know that PTC 124 is now in phase III trials for adults over 18. Thus far it has shown great promise for any CFer who has at least one mutation with an X at the end. I don't know if/when it will go to trial for younger patients. If you google it you will find that it is also showing promise for helping duchene musular dsytrophy. I believe it helps to fix the "stop" in the genetic coding and thus gets the body to read through and create the protein that was not working. So in CF it with get the CFTR to work properly and the idea would be that with the drug (daily) a patient would be more like a carrier.
I hope this helps.
I hope this helps.
MargaritaChic
New member
That would be wonderful! The genetic person at our CF clinic says the research that is being done on the X mutations is very promising.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
MargaritaChic
New member
That would be wonderful! The genetic person at our CF clinic says the research that is being done on the X mutations is very promising.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
MargaritaChic
New member
That would be wonderful! The genetic person at our CF clinic says the research that is being done on the X mutations is very promising.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
MargaritaChic
New member
That would be wonderful! The genetic person at our CF clinic says the research that is being done on the X mutations is very promising.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
MargaritaChic
New member
That would be wonderful! The genetic person at our CF clinic says the research that is being done on the X mutations is very promising.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
I found an article that says the drug may be available as soon as 2009.
My daughter is less than 1 month old now, so this gives me hope for her future.
You can go to PTCbio.com for updates. I read where they want to try it for pediatrics this year. My son has an X mutation so I'm hoping to get him into a trial. I believe one is going to be done at John Hopkins. 50 sites in the US all together. We go to DuPont in DE and his Dr. said if they don't participate and another site near us will we can get him into that clinic for the clinical trials. I'm excited for this!
laurie
laurie
You can go to PTCbio.com for updates. I read where they want to try it for pediatrics this year. My son has an X mutation so I'm hoping to get him into a trial. I believe one is going to be done at John Hopkins. 50 sites in the US all together. We go to DuPont in DE and his Dr. said if they don't participate and another site near us will we can get him into that clinic for the clinical trials. I'm excited for this!
laurie
laurie
You can go to PTCbio.com for updates. I read where they want to try it for pediatrics this year. My son has an X mutation so I'm hoping to get him into a trial. I believe one is going to be done at John Hopkins. 50 sites in the US all together. We go to DuPont in DE and his Dr. said if they don't participate and another site near us will we can get him into that clinic for the clinical trials. I'm excited for this!
laurie
laurie
You can go to PTCbio.com for updates. I read where they want to try it for pediatrics this year. My son has an X mutation so I'm hoping to get him into a trial. I believe one is going to be done at John Hopkins. 50 sites in the US all together. We go to DuPont in DE and his Dr. said if they don't participate and another site near us will we can get him into that clinic for the clinical trials. I'm excited for this!
laurie
laurie
You can go to PTCbio.com for updates. I read where they want to try it for pediatrics this year. My son has an X mutation so I'm hoping to get him into a trial. I believe one is going to be done at John Hopkins. 50 sites in the US all together. We go to DuPont in DE and his Dr. said if they don't participate and another site near us will we can get him into that clinic for the clinical trials. I'm excited for this!
laurie
laurie