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Usually the saftey trial is done on healthy adults rather than on children.
jenn said:does that mean that they may consider an american child for the current trial? i got an "out of office reply" so i hope they will reply when they are back, it said july1st.
Mindy said:They told me at the time that it was a safety trial only - didn't expect efficacy, and didn't want people to move from home just to verify safety.
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.
jenn said:I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
Does anyone know how I can get information on Charley's fund? Maybe a phone number or email. Thanks so much.
Julie Hathaway said:My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.
jenn said:I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
An idea for consideration if your sons are nonambulatory when the trial starts is when our son Jacob was denied PTC 124 the head of the neuro pharm div of the FDA recommended a protocal exception. A child in a trial with a protocal exception is treated the same as all the others but they do not include outcome measures when they drug company send its report to the FDA. Unfortunately PTC would not go along with the FDA. The FDA actually encourages expanded access ie compassionate use or a protocal exception in rare diseases such as duchenne so there are more on the drug in a controlled setting before it goes to market. I found the FDA to bend over backwards trying to help Jacob get on PTC 124. Feel free to email me if you have any questions, cgunval@gvtel.com
Julie Hathaway said:Does anyone know how I can get information on Charley's fund? Maybe a phone number or email. Thanks so much.
Julie Hathaway said:My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.
jenn said:I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
My son will be 7 in July. He has a deletion on exon 51. I don't think he would benefit from this trial. Am I correct? I just returned from the conference and the news about exon skipping is very exciting. Kathy Busby from the UK stated that trials would be taking place in the U.S. I think my son needs 50 or 52 skipped. Blessings to all of you.
jenn said:I actually have a cousin living in london for another year or so...if I hear anything from the trial folks I can provide them a local address,of course we'd rent once we got there, but at least that's a start. Yes, I think we should make our son's known as candidates for trials, older boys won't have a chance by the time trials hit the us. I don't know how I feel about the chance that austin won't get the drug, but I KNOW he won't if we stay here and wait. Why would they use a placebo if the safety study is done, can't they just do a natural course study of duchenne?
Hello all,
I think we all need to be careful about a couple of things:
1. These trials are very preliminary right now. The clinic has not met with the FDA to establish protocol, final qualifications, etc. I don't think we should speculate on any of that information until it is finalized so that we don't spread rumors. Lindsay has said that she'll be in touch with the families personally with any developments, so if you hear anything from anyone other than her, don't take it as the final word.
2. We need to be respectful of the people planning these trials, including being careful about posting emails from them, etc. without their permission. Trials like these are very fragile in the planning stages, and agitating in any way doesn't help the process.
IHere are the facts of the trial from Lindsay:
it is systemic, not intramuscular
we don't know dosage
there are risks involved in participating and no guarantees
'm not trying to be the cop here, but I don't want Mendell's office bombarded with controversy and conflict when they're just trying to assess interest in participation.
Thanks,
Mindy
i have looked at the web site, but it doesn't say anywhere that there is a trial in columbus, only london... also is this just a safety trial or will it actually produce results( hopefully)? i sent our info, i have 2 boys, we had thought they had a deletion of 51, but it is 52, so i think they qualify for this.
Hey Jenn,
My son has a deletion of 52 and I am desperately trying to find someone trying 52. Columbus is doing 51 at present...if you hear, please let me know. thanks
JoAnne
jenn said:i have looked at the web site, but it doesn't say anywhere that there is a trial in columbus, only london... also is this just a safety trial or will it actually produce results( hopefully)? i sent our info, i have 2 boys, we had thought they had a deletion of 51, but it is 52, so i think they qualify for this.
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