I just read in the on-line Quest Magazine for MDA, that Dr. Mendell's AVIPharma Exon Skipping trials are beginning in March of 2010. I signed up for Nicolas to be a part of the trials back in April and have every now and then emailed the coordinator to get updates. He did confirm that it would be taking place at that time, and right now they are getting their protocols together and should have more information in a couple weeks.

Here's my dilemma No. 1. We see Dr. Wong, and she too will be starting clinical trials for the Netherlands exon skipping drug trial (not the same as Dr. Mendell), but I have no timeframe from her of when her trials will begin. I was told last year that they would start summer of this year.... It's almost Christmas. I want something now and don't want to wait any longer.... first come first serve I guess.

My other dilemma is that my husband and I both work in Illinois and the trial is on-site in Columbus Ohio for 12 weeks, and then about 5 follow up visits, every couple weeks after that. This will of course cost me to lose my job, and in this economy, we absolutely cannot afford that. Of course my heart and thoughts are with my son and no matter what, if he is accepted into the trial, we will be there, job or no job. This is definitely more important, but I can't help but think about the future of not having a job... most likely having a home foreclose on us (we are definitely not wealthy), and living with relatives (really not close with alot of them), etc. etc. etc. Also, not having enough $$ to afford to buy Nicolas his deflazacort, and supplements, and not to mention still paying for this years visit to see Dr. Wong.
I guess I just wanted to throw my worries out there. Hopefully it's all for the best. Is anyone else up for the trials or know about Dr. Wong's???

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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.



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.
I agree - register with everyone!!! With DMD - one can never make assumptions.

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.



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.
Definitely Brian. My son is registered with DuchenneConnect. What I wanted to say is that we need to make sure that a boy registered with DuchenneConnect IS in the Treat-NMD registry and not be left out due to any problems. I hope that DuchenneConnect fixed the problems noted by parents in the link I provided and is functioning as accurately as possible.

Prosensa/GSK's trial is a huge step in the right direction for all our sons and we need to insure that all boys qualified have the opportunity to be contacted for the trial.

Also, as we all know, AVI/Jerry Mendell's trial is not using DuchenneConnect. Interested parents should contact the research coordinator directly.

Brian Denger said:
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.
http://www.actionduchenne.org/jsp/uploaded_files/documents/newslett...

News of collaboration between Prosensa, the Dutch biopharmaceutical company and GSK is a
‘win win win’ for both the companies and Duchenne patients, says Luc Dochez, Vice President Business
Development at Prosensa.

The new alliance recently announced in October between GSK and Prosensa is a “win win win” for all
parties. Established under GSK’s Centre of Excellence for External Drug Discovery (ceedd), the alliance
means that the two companies will be working on an exclusive collaboration for the development and
commercialisation of RNA based therapeutics for DMD. With the new agreement, GSK has a worldwide licence to develop
Prosensa’s lead compound, PRO051, designed to treat DMD by skipping exon 51 of the dystrophin gene. Working together, we will continue to progress the further development of PRO051 - we have
already begun preparations for a Phase III study which we expect will start in early 2010.

The best of both worlds for DMD patients
For DMD patients the alliance will bring benefits as we work
together to develop and bring to patients more therapies in shorter
timescales to treat DMD. We are committed to getting treatments to
patients as quickly as possible, but it is important that we follow the
established safety paths for commercial development.
The alliance is a complimentary match of skills and experience that
will enable us to move much faster and broader in a structured way.

GSK’s significant financial resources and operational expertise of
large scale pharmaceutical development, matched with our niche
experience in exon skipping and Duchenne will enable us to take
drugs faster through the development path.
Extending the studies to treat more patients
While RNA-based therapeutics (antisense oligonucleotides inducing
exon skipping) are among the most promising therapies for DMD,
different mutations in the gene require different oligonucleotide
drugs. PRO051 may provide treatment for 13% of boys with
Duchenne.
With this new alliance GSK has exclusive options to licence three
more RNA-based compounds targeting additional DMD exons. One
such option includes PRO044, which targets the skipping of exon
44. At Prosensa we are expecting to start a Phase I/II study for
PRO044 before the end of this year (2009)
. On successful
completion of this study, GSK will then have the option to proceed
with the further development and production of the compound.
We are excited by this new alliance, which will not only benefit boys
with Duchenne in Europe and the US, but will also open up new
opportunities extending to other regions like Japan and South
America.
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.
Michelle,

http://www.acfarma.com/flacort.htm

It´s only in spanish but please ask someone to help you translating, I do believe we need to help each other to help our kids, please take advantage of what we have in Peru.

I took some samples to Leslie Guzmán from UPA latin american site when we saw her on our visit to Dr. Wong.

PLease let me know if this is helpfull.

JP.

Michelle Gonzales said:
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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