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The press release only mentions 51 and 44. I don't know if the other two have been officially announced.
I would guess the other two will be 45 and 53, which would then give them coverage for the 4 most commonly needed exon skips. Just a guess on my part, though.
Prosensa is gearing up for their exon 51 skipping trial - they are hoping to begin some time in this spring 2010. The information is not concrete as of yet, but what's filtering down is that they will be looking for upwards of 150 participants for a drug/placebo trial method to ensure good results. Those kids getting the placebo - if the drug works, will receive it in the extension study at the trials end. They maintain that all participants will be taken from the Treat-NMD GLobal database. If you are not signed up - please take the time to get your son registered if exon 51 skipping will help. www.treat-nmd.eu.
Keith Van Houten said:The press release only mentions 51 and 44. I don't know if the other two have been officially announced.
I would guess the other two will be 45 and 53, which would then give them coverage for the 4 most commonly needed exon skips. Just a guess on my part, though.
Hello.
I want to clarify how recruitment works in planned clinical trials. Besides participant recruitment, registries are often used to help coordinators plan studies (Determine areas where many patients live, etc.) and announce study and trial opportunities. Study coordinators may use other resources for participant recruitment. It is possible that participants will be recruited from participating clinics. The key is knowing the participants mutation type and meeting other eligibility requirements referred to as inclusion and exclusion criteria. At this time there is no announcement concerning these trials, so recruitment methods are unknown.
DuchenneConnect (https://www.duchenneconnect.org) is a partner in the TREAT-NMD program and TREAT-NMD works through each country's registry program. In the United States, if your son is registered with the DuchenneConnect registry, has his genetic test results on file and have updated the profile in the past 12 months then he is included in the TREAT-NMD Global Registry program. There is no need to enroll in another registry.
Once an announcement for recruitment is available for these planned trials, methods for recruitment will be available.
I hope this is helpful.
Brian Denger
irishgirl said:Prosensa is gearing up for their exon 51 skipping trial - they are hoping to begin some time in this spring 2010. The information is not concrete as of yet, but what's filtering down is that they will be looking for upwards of 150 participants for a drug/placebo trial method to ensure good results. Those kids getting the placebo - if the drug works, will receive it in the extension study at the trials end. They maintain that all participants will be taken from the Treat-NMD GLobal database. If you are not signed up - please take the time to get your son registered if exon 51 skipping will help. www.treat-nmd.eu.
Keith Van Houten said:The press release only mentions 51 and 44. I don't know if the other two have been officially announced.
I would guess the other two will be 45 and 53, which would then give them coverage for the 4 most commonly needed exon skips. Just a guess on my part, though.
How do we check to verify that indeed registering with DuchenneConnect is all that is needed to be part of the Treat-NMD registry?
Several problems with DuchenneConnect were reported by parents here: http://community.parentprojectmd.org/profiles/blogs/exon-skipping-w...
I think that we need to be certain that our sons are not missing such opportunities due to problems with DuchenneConnect especially when it is so easy to register them in the Treat-NMD registry ourselves. Prosesa is using Treat-NMD for their current trial. This was specified by them several times at conferences/meetings.
How do we check to verify that indeed registering with DuchenneConnect is all that is needed to be part of the Treat-NMD registry?
Several problems with DuchenneConnect were reported by parents here: http://community.parentprojectmd.org/profiles/blogs/exon-skipping-w...
I think that we need to be certain that our sons are not missing such opportunities due to problems with DuchenneConnect especially when it is so easy to register them in the Treat-NMD registry ourselves. Prosesa is using Treat-NMD for their current trial. This was specified by them several times at conferences/meetings.
Brian Denger said:Hello.
I want to clarify how recruitment works in planned clinical trials. Besides participant recruitment, registries are often used to help coordinators plan studies (Determine areas where many patients live, etc.) and announce study and trial opportunities. Study coordinators may use other resources for participant recruitment. It is possible that participants will be recruited from participating clinics. The key is knowing the participants mutation type and meeting other eligibility requirements referred to as inclusion and exclusion criteria. At this time there is no announcement concerning these trials, so recruitment methods are unknown.
DuchenneConnect (https://www.duchenneconnect.org) is a partner in the TREAT-NMD program and TREAT-NMD works through each country's registry program. In the United States, if your son is registered with the DuchenneConnect registry, has his genetic test results on file and have updated the profile in the past 12 months then he is included in the TREAT-NMD Global Registry program. There is no need to enroll in another registry.
Once an announcement for recruitment is available for these planned trials, methods for recruitment will be available.
I hope this is helpful.
Brian Denger
irishgirl said:Prosensa is gearing up for their exon 51 skipping trial - they are hoping to begin some time in this spring 2010. The information is not concrete as of yet, but what's filtering down is that they will be looking for upwards of 150 participants for a drug/placebo trial method to ensure good results. Those kids getting the placebo - if the drug works, will receive it in the extension study at the trials end. They maintain that all participants will be taken from the Treat-NMD GLobal database. If you are not signed up - please take the time to get your son registered if exon 51 skipping will help. www.treat-nmd.eu.
Keith Van Houten said:The press release only mentions 51 and 44. I don't know if the other two have been officially announced.
I would guess the other two will be 45 and 53, which would then give them coverage for the 4 most commonly needed exon skips. Just a guess on my part, though.
Hello Ofelia,
There are many reasons to participate in a registry in the US. Study coordinators use these tools to assess patient populations in a given area ensuring trials are located where more participants can be found. This makes sense to avoid travel not only as an expense, but also as a burden to families. The DuchenneConnect registry collects all the required information to participate as a partner global registry and additional information based on what the advisers deem appropriate for natural history and other criteria. Researchers hoping to learn more about this population and the more patients registered, the more robust the data.
If you go to the TREAT-NMD website it is posted that families should register in the registry available in your country:
http://www.treat-nmd.eu/patients/patient-registries/national-regist...
Additionally, going to the link for registries there is the statement:
"Please note that we encourage you to register only once, since the information from the national registries is transferred to the TREAT-NMD global database and there is therefore no benefit in registering in more than one country."
Going to the US registries link there are two registries available. DuchenneConnect is the largest and is designed to benefit research and provide families with much needed information. Additionally there is a genetic counselor available to help families with questions and concerns for genetic testing.
There were initial problems with posting information from clinicaltrials.gov, yet this has been corrected.
Brian Denger
Ofelia Marin said:How do we check to verify that indeed registering with DuchenneConnect is all that is needed to be part of the Treat-NMD registry?
Several problems with DuchenneConnect were reported by parents here: http://community.parentprojectmd.org/profiles/blogs/exon-skipping-w...
I think that we need to be certain that our sons are not missing such opportunities due to problems with DuchenneConnect especially when it is so easy to register them in the Treat-NMD registry ourselves. Prosesa is using Treat-NMD for their current trial. This was specified by them several times at conferences/meetings.
Michelle,
I live in Lima Peru, we have liquid deflazacort OTC without prescription here in Lima, the name is Flacort, check out the web and if you need it for your son trust me it is very cheap and from local recognized Lab.
best wishes
JP.
PS: the trial from GSK - Prosensa will start " early 2010" tht´s what they say.
Pray thta the kids muscles show enough evidence of strengthness...maybe this will speed up the approval.
Thank you Juan Pedro! Is there a web address that you have for Flacort?
JUAN PEDRO ARBULU said:Michelle,
I live in Lima Peru, we have liquid deflazacort OTC without prescription here in Lima, the name is Flacort, check out the web and if you need it for your son trust me it is very cheap and from local recognized Lab.
best wishes
JP.
PS: the trial from GSK - Prosensa will start " early 2010" tht´s what they say.
Pray thta the kids muscles show enough evidence of strengthness...maybe this will speed up the approval.
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