This discussion is to hopefully stay connected with other families of boys who participated on the Phase 2a (38 boys in three sites), and to communicate with all the DMD community the progress of our boys while on PTC124.
My son Daniel Castro (10 years old) participated in the 28 days clinical trial of PTC124 along with other 37 boys a couple of years ago. Since, we have been patiently wating for the extension study. Thank God as of last week, we got the confirmation from Dr. Finkel's team in Philadelphia that everything is ready to go, and they are asking ALL the families of the boys who participated to request the weeks in which they would prefer to go to start the new study. If you are one of those families and you haven't gotten in contact with your hospital in which you did the original trial, please contact them ASAP to get this going for our boys. Great news is that our boys will be taken PTC124 for almost 2 years. They will need to get muscle byopsies again, but that is an small price to pay for the potential benefit we all expect to get out of it.
I encourage parents and families in general of these boys to use this discussion to talk about anything related to the development of this new study, and to report progress, complications, experiences, syntoms, differences noticed while in treatment etc. I think it would be good not only for those 38 families, but hopefully for ALL the kids with non-sence mutation who are participating on the new 2b study and even for all other DMD kids with other type of mutations since PTC therapeutics is working on a new medicine for ALL the cases of DMD.
God bless you ALL.
Jose, Angie, Daniel and the Castro Gang.

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Angela, you are welcome. Hopefully your son is taking PTC124 and very soon you will star noticing improvements on him. Please keep us posted on any progress. I will do the same when my son starts taking PTC124 again and hopefully we notice improvements.
God bless
I feel we are being patient, especially now that we are very distracted and busy with school. I know they are straight out and that the scheduling is very difficult. I'm just very much into communication, so wish this were better. We did hear from Teesha the other day, so I know things are moving along. I'm fine with traveling and agree it's a small price; just so happy they arrange and take care of it. We'll be thinking about your son and hope he regains his mobility. The news from parents who have started the drug in Utah already is all good so far. We're scheduled to go down to Gainesville for the MRI trial in early October. Hope you are fairing well with all the hurricanes lately. Best wishes, Joanne


dacas01 said:
Please ignore my previous post i made a mistake..... Sorry

Joanne, we are very much in the same situation. We are still waiting. We got his echo and EKG a couple of days ago. No dates just waiting. I want to keep possitive and think that we have awaited over 2 years jsut a few more weeks doesn't look that long. My son is 10 and he stoped walking about 4 or 5 months ago. Trust me we want this yesterday, but unfortunately is out of our control. I don't want to get upset with Dr. Finkell and his team since i feel they are doing things as fast as the protocol permits. All i can tell you, coming from someone in the exact same position you are, lets try to be a little bit more patient, it will happen before we know it. We live in South Florida and will have to do a lot of traveling for the procedures, but that is a very small price. No blood work done as of today yet. As soon as i know more i will post it here.
Best Regards
Thanks Joanne, same thing here Teesha has been comunicating with me as well. We are almost there. As far as the hurricanes we have been lucky so far. Hoping it continues that way for the rest of the season. Do you know anyone of the 38 families from Utah who are already taking PTC124 as part of the pararell staudy??? If so, do you think they would mind joining this thread and start sharing a little bit of their progress?? The purpose here is to be able to share our experiences, progress, side effects (hopefully none) and whatever else we can to help each other looking for the best interest of not only our kids but the entire MD community.
Where do you guys live???
God bless
Jose
Hi Jose,

I have been following the Utah parents through their caring bridge sites.
http://www.caringbridge.org/visit/anthonycastle and http://www.caringbridge.org/visit/jackknight

I think it's a great idea to invite them to join this forum. I don't have any idea whether anyone has started at the Ohio site. Dr. Finkel explained that Utah was able to get started faster because they are not doing the 2b study. Do you know others in the study we can personally invite using the "Invite More" button on the right of the posts?

We live in northern Vermont, outside of Burlington. We're holding on to every last bit of warm, sunny weather we can, until the weather really starts to change. We get the remnants of Ike tomorrow.
We started a caring bridge site for Adam too to help keep family and friends up to date: http://www.caringbridge.org/visit/adamwechsler
Joanne



dacas01 said:
Thanks Joanne, same thing here Teesha has been comunicating with me as well. We are almost there. As far as the hurricanes we have been lucky so far. Hoping it continues that way for the rest of the season. Do you know anyone of the 38 families from Utah who are already taking PTC124 as part of the pararell staudy??? If so, do you think they would mind joining this thread and start sharing a little bit of their progress?? The purpose here is to be able to share our experiences, progress, side effects (hopefully none) and whatever else we can to help each other looking for the best interest of not only our kids but the entire MD community.
Where do you guys live???
God bless
Jose
Joanne, thanks for sharing. WOW i went to Anthony's page and.... God am i excited. I pray this might be the outcome for ALL 38 kids on the study. This is crazy. You made my day. I also went to your page and i got to tell you, you have a beautiful family. God bless you ALL. I will create a page for Daniel and will share the address after done. I will attempt to contact them and invite them to post here or at least get their permission to share here some of their posts from their sites, that way other people might see it here as well. Anyways, thanks again.
Jose
I just sent an e-mail to Jill about joining this group. I thought you might enjoy her latest journal on caringbridge. And I'm glad you enjoyed our pictures. The Caring Bridge sites are fun. It's therapeutic for me (and for Adam) to write and share. It definitely helps us deal with Duchenne. Like most of us, this is definitely the biggest challenge my family has ever had to face.
Joanne


dacas01 said:
Joanne, thanks for sharing. WOW i went to Anthony's page and.... God am i excited. I pray this might be the outcome for ALL 38 kids on the study. This is crazy. You made my day. I also went to your page and i got to tell you, you have a beautiful family. God bless you ALL. I will create a page for Daniel and will share the address after done. I will attempt to contact them and invite them to post here or at least get their permission to share here some of their posts from their sites, that way other people might see it here as well. Anyways, thanks again.
Jose
Awesome, lets hope they join and since they are leading the pack we might have a lot of things comming from them we all might benefit and learn from. Also, yes i agree 100 % on the remark that DMD is the bigest challenge we have to face, but things are looking very promising now. I think we are living in exciting times as far as the new developments, the technology not only from the scientist perspective but from the communication as well. It is amazing that thru the internet we now can share info like never before and be able to help each other by sharing our experiences etc. Go bless, Jose
So, when I went to the the PPMD home page, then the Groups Page, then clicked on 2a Extension Group, the page opens up, but it doesn't look like there is any discussion going on. I'm so not tech savvy. Try it and you will see what I mean. This would not feel inviting to me if I was new. Joanne

dacas01 said:
Awesome, lets hope they join and since they are leading the pack we might have a lot of things comming from them we all might benefit and learn from. Also, yes i agree 100 % on the remark that DMD is the bigest challenge we have to face, but things are looking very promising now. I think we are living in exciting times as far as the new developments, the technology not only from the scientist perspective but from the communication as well. It is amazing that thru the internet we now can share info like never before and be able to help each other by sharing our experiences etc. Go bless, Jose
Humm. You are right. I think there is an issue with the site, because you can actually see our current discussion when you cklick on the discussion forum under reseach, and PTC124 Phase 2A Extension Study. On Monday i will contact the tech in PPMD and see if we are doing something wrong or in fact there is an issue with the site, that it isn't attaching the discussions to the groups. Regards
Jose
I think I know why. We are talking under the Discussion Board/Research/2a; rather than within the Group of 2a. Again, no techie, but I think there are two different places to be having conversations. Right now we are in the "discussion forum", which is more public than talking within the group, which is a "membership" thing. Joanne


dacas01 said:
Humm. You are right. I think there is an issue with the site, because you can actually see our current discussion when you cklick on the discussion forum under reseach, and PTC124 Phase 2A Extension Study. On Monday i will contact the tech in PPMD and see if we are doing something wrong or in fact there is an issue with the site, that it isn't attaching the discussions to the groups. Regards
Jose
Hi Everyone,

I Utah is currently running. My son and husband will be traveling out there this week to begin the trial. We all have been waiting a long time for this. We are very excited about the prospect to continue the trial.

Joanne Wechlser said:
Hi Jose and gang,
Just checking in again. Do you have dates? It's Sept 10th now and we're still waiting. I've been staying in touch with them, but still not getting anything definitive. So yes, my patience is getting tired, as now we are into school and a million other activities. You're right we are lucky. We had my son's echo and ekg done in July in anticipation of an August date. Are you in the Philly area or will you be traveling? Have you started with the other screening with the blood work?
We're hanging in there, but a little anxious. My son is 12 already.
Joanne
Hi Christine,
We'll be thinking about you guys. I'm still waiting on my schedule, but we are slated to start soon in Philly. Meanwhile, we are headed to Florida for the MRI portion Oct. 1-3. We're excited about the prospect too, but a little nervous. Our school agreed to provide Adam a laptop, so he can feel more connected while we are traveling and log on to his homework pages. I'll feel better once we have some details ironed out. We'll look forward to hearing how it goes for you all. Best, Joanne

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