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PTC124
- Thread starter bblond
- Start date
<blockquote>Quote<br><hr>My son finished the trial on December 27th- he participated in Birmingham Alabama. It definitely showed promise. They are also getting good results in Israel. At our last appt there were two more people enrolled in Bham- may be more now. I dont think anymore of the US sites were up and going at that time- cant wait for this phase to be finished and on to the next. Susan <hr></blockquote>
Susan,
I am dying for details. I live in California and there are no trials on the West Coast. Otherwise, I would participate. What happened with your son when on the drug? Did pft's go up? sputum dissapear? etc. Does he think it cured him? When you say it shows promise and that they are getting good results, can you give me some details? I am so hopefull for this to be a silver bullet. Feel free to contact me offline as well at bblond@gmail.com
Thanks!!
Brian
Susan,
I am dying for details. I live in California and there are no trials on the West Coast. Otherwise, I would participate. What happened with your son when on the drug? Did pft's go up? sputum dissapear? etc. Does he think it cured him? When you say it shows promise and that they are getting good results, can you give me some details? I am so hopefull for this to be a silver bullet. Feel free to contact me offline as well at bblond@gmail.com
Thanks!!
Brian
<blockquote>Quote<br><hr>My son finished the trial on December 27th- he participated in Birmingham Alabama. It definitely showed promise. They are also getting good results in Israel. At our last appt there were two more people enrolled in Bham- may be more now. I dont think anymore of the US sites were up and going at that time- cant wait for this phase to be finished and on to the next. Susan <hr></blockquote>
Susan,
I am dying for details. I live in California and there are no trials on the West Coast. Otherwise, I would participate. What happened with your son when on the drug? Did pft's go up? sputum dissapear? etc. Does he think it cured him? When you say it shows promise and that they are getting good results, can you give me some details? I am so hopefull for this to be a silver bullet. Feel free to contact me offline as well at bblond@gmail.com
Thanks!!
Brian
Susan,
I am dying for details. I live in California and there are no trials on the West Coast. Otherwise, I would participate. What happened with your son when on the drug? Did pft's go up? sputum dissapear? etc. Does he think it cured him? When you say it shows promise and that they are getting good results, can you give me some details? I am so hopefull for this to be a silver bullet. Feel free to contact me offline as well at bblond@gmail.com
Thanks!!
Brian
Ok- I will try to explain what they found. Its hard for me to understand exactly but I will try- he was on a lower dose of the drug for two weeks, off two, back on a higher dose for two. He was in the hospital 4 times- once before lower dose , then after, before higher dose and again after. They did nasal pds to measure the results and took nasal scrapings. The pd after the first dose showed what the dr describe as more like normal than cf but not completely normal. After the higher dose it showed the same and we were a little disappointed but when they got the nasal scrapings back they were very excited. What they were measuring- again I cant get it completely straight in my brain-Before Will took the meds he measured a 3- The Dr measured himself to have something to compare to and he measured a 52. After Will took the higher dose of med he measured a 26. One nurse said that he could very well function with that. Again-this may not be real clear but I know they were excited. They kept trying to remind me that this phase was mainly to test for safety and figure out the best dosage. Will didnt have any bad side effects- his throat hurt about a minute after he drank the meds and he had some burning when he urinated the first time in the morning. I do think he coughed less and felt better when he took the med- that is just me as a mom- he really does very well anyway. They didnt remeasure pfts. They hope the next phase will be about in a yr and involve more and younger patients with longer time on the med. If you are interested and can take the time to be in the study they were very good with reimbursement for travel and hotel expenses for me. I think there is a study in Colorado. I hope this is helpful to you -Susan
Ok- I will try to explain what they found. Its hard for me to understand exactly but I will try- he was on a lower dose of the drug for two weeks, off two, back on a higher dose for two. He was in the hospital 4 times- once before lower dose , then after, before higher dose and again after. They did nasal pds to measure the results and took nasal scrapings. The pd after the first dose showed what the dr describe as more like normal than cf but not completely normal. After the higher dose it showed the same and we were a little disappointed but when they got the nasal scrapings back they were very excited. What they were measuring- again I cant get it completely straight in my brain-Before Will took the meds he measured a 3- The Dr measured himself to have something to compare to and he measured a 52. After Will took the higher dose of med he measured a 26. One nurse said that he could very well function with that. Again-this may not be real clear but I know they were excited. They kept trying to remind me that this phase was mainly to test for safety and figure out the best dosage. Will didnt have any bad side effects- his throat hurt about a minute after he drank the meds and he had some burning when he urinated the first time in the morning. I do think he coughed less and felt better when he took the med- that is just me as a mom- he really does very well anyway. They didnt remeasure pfts. They hope the next phase will be about in a yr and involve more and younger patients with longer time on the med. If you are interested and can take the time to be in the study they were very good with reimbursement for travel and hotel expenses for me. I think there is a study in Colorado. I hope this is helpful to you -Susan
Christian,
Mark was DX'd in 1982/1983ish time as well. There was always mention of his mutations being Double Delta F508 but we could never find it in his medical records. We told the doctors we were curious (we also told them we wanted the information now that we are married, considering a family, want to identify Mark's mutations so we can test the baby, just to be sure) and they ordered a test of the 25-89 most common mutations. Mark is a Double D F 508. It's a blood test that can be sent to a local laboratory, I am guessing since you were Dx'd in the 80's that you probably have common mutations so you wouldn't need an extended panel (which is more expensive and usually gets denied anyways by ins. co's.)
Good luck, when you get tested, let us know what kind of mutant you are <img src="i/expressions/face-icon-small-smile.gif" border="0">
Mark was DX'd in 1982/1983ish time as well. There was always mention of his mutations being Double Delta F508 but we could never find it in his medical records. We told the doctors we were curious (we also told them we wanted the information now that we are married, considering a family, want to identify Mark's mutations so we can test the baby, just to be sure) and they ordered a test of the 25-89 most common mutations. Mark is a Double D F 508. It's a blood test that can be sent to a local laboratory, I am guessing since you were Dx'd in the 80's that you probably have common mutations so you wouldn't need an extended panel (which is more expensive and usually gets denied anyways by ins. co's.)
Good luck, when you get tested, let us know what kind of mutant you are <img src="i/expressions/face-icon-small-smile.gif" border="0">
Christian,
Mark was DX'd in 1982/1983ish time as well. There was always mention of his mutations being Double Delta F508 but we could never find it in his medical records. We told the doctors we were curious (we also told them we wanted the information now that we are married, considering a family, want to identify Mark's mutations so we can test the baby, just to be sure) and they ordered a test of the 25-89 most common mutations. Mark is a Double D F 508. It's a blood test that can be sent to a local laboratory, I am guessing since you were Dx'd in the 80's that you probably have common mutations so you wouldn't need an extended panel (which is more expensive and usually gets denied anyways by ins. co's.)
Good luck, when you get tested, let us know what kind of mutant you are <img src="i/expressions/face-icon-small-smile.gif" border="0">
Mark was DX'd in 1982/1983ish time as well. There was always mention of his mutations being Double Delta F508 but we could never find it in his medical records. We told the doctors we were curious (we also told them we wanted the information now that we are married, considering a family, want to identify Mark's mutations so we can test the baby, just to be sure) and they ordered a test of the 25-89 most common mutations. Mark is a Double D F 508. It's a blood test that can be sent to a local laboratory, I am guessing since you were Dx'd in the 80's that you probably have common mutations so you wouldn't need an extended panel (which is more expensive and usually gets denied anyways by ins. co's.)
Good luck, when you get tested, let us know what kind of mutant you are <img src="i/expressions/face-icon-small-smile.gif" border="0">
princessjdc
New member
<blockquote>Quote<br><hr><i>Originally posted by: <b>bblond</b></i><br>While it is true that the drug would just fix one of the mutations, that is all that is needed to cure CF. If we just had one mutation then we would be a carrier and not have full blown CF.<hr></blockquote>
so then in that case I wouldnt have cf at all anymore then, cause both my mutations are G542x, and you guys are talking about the mutations with an x at the end right?
so then in that case I wouldnt have cf at all anymore then, cause both my mutations are G542x, and you guys are talking about the mutations with an x at the end right?
princessjdc
New member
<blockquote>Quote<br><hr><i>Originally posted by: <b>bblond</b></i><br>While it is true that the drug would just fix one of the mutations, that is all that is needed to cure CF. If we just had one mutation then we would be a carrier and not have full blown CF.<hr></blockquote>
so then in that case I wouldnt have cf at all anymore then, cause both my mutations are G542x, and you guys are talking about the mutations with an x at the end right?
so then in that case I wouldnt have cf at all anymore then, cause both my mutations are G542x, and you guys are talking about the mutations with an x at the end right?