Hi Sharon, Thanks for all the help that PPMD has been offering. My son (4.5 years old) has regular duplication of exon 51-55. Can you please help me find any clinical trials that he can participate in. Thanks, Satish
hi sharon i want would like to say thank you very much for working so hard to help our boys that one day we will find a cure . i have been seeing lots of comments about the drug avi how is going to help our boys and how soon will get it fda approve. thanks kimmy
thank you sharon that explain alot i would to be able to watch the web on wed but i am on dial up very slow but am great ful it still work. i am also very exctied that about the ophan drug. will that help alot of our boys. will it help them live longer.kimmy
hi sharon my name is kimmy watters andi have a question, the sweet young boy brady 14 lives ca. what is the fat emoby sorry i do not spell well. he was such a sweet kid very handsome as well thank you kimmy watters
Thank you so much for all your supports and suggestions. It helps me and my family so much, especially about the heart specialist for my brother. I'm very appreciated for your kindness. If there's a chance that the community coming abroad to Thailand, that would be very great as well because there're actually alot of people who face difficulty with the Duchenne. Moreover, once again thank you so much. And, if there're any more problems or questions, would you mind to ask u again ??.
Dear...Sharon, My brother, he cannot walk anymore, cannot move any parts of his body except he can still shew some food, talk as normal people, and move his head. But he can't eat alot as others. Also, he cough alots. He needs to use the respirator all the time otherwise he's hard to breath by hisown. Unfortunately, there are not any treatments in Thailand. Therefore, he hasn't taken any medicine or done anything about medication. Several times, he can't breath so my parents take him to the hospital for getting some supplimentaly foods. Accordingly, What should i do??I really want him to do some treatments or make him be better.. Thank you for your kindness
My brother, he cannot walk anymore, cannot move any parts of his body except he can still shew some food, talk as normal people, and move his head. But he can't eat alot as others. Also, he cough alots. He needs to use the respirator all the time otherwise he's hard to breath by hisown. Unfortunately, there are not any treatments in Thailand. Therefore, he hasn't taken any medicine or done anything about medication. Several times, he can't breath so my parents take him to the hospital for getting some supplimentaly foods. Accordingly, What should i do??I really want him to do some treatments or make him be better.. Thank you for your kindness
If you are interested, please join in the discussion about electrostim treatment at South Texas Innovative Medicine Discussion Forum. My daughter has RSD and went to Dr Rhodes and STIM. The "treatment" didn't work at all for her. The STS has been on the market for almost a DECADE! I'm suspicious that there's never "time" to assess the success rate data...
Hey Sharon, Just wondering if you could give feedback on Acceleron and their recent infusion of $$ from Shire @ 480mil. Our son has a duplication of 54-57 and exon skipping is not an option b/c of the duplication. I was watching Biomarin but they dropped out. Now Acceleron. Biomarin was trying to turn on utrophin vs. Acceleron is trying to work with myostatin. Any other things I should be watching for duplications? Thanks Char Burke - mom to Will age 8.
Thank you so much for helping out our community. I see how hard you work to find us the information we need. Am I the only one here on this site, that just can't take all this negativity anymore? Some of these discussions just make me sick. Currently there is much discussion as to the GSK/Prosensa trials. Do you know what is really going on with the trials? I just can't take it anymore, all this talk of the FDA willing to kill our sons. I just can't look at it like that, it pains me to hear people talk this way. Is there anything positive we can do? Is there something we don't know? If you maybe could just clarify for us that would be great. I feel that another attack is brewing and some of us really don't need to hear such shocking statements. I feel bad like you have been attacked many times, just trying to find the information we need.
AVI-4658 is designed to cause cells to snip out a small piece of DNA called “exon 51” to allow boys with certain types of deletions in their dystrophin genes (deletions of exons 45-50, exons 47-50, exons 48-50, exons 49-50, exon 52 and exons 52-63) to be able to make a smaller, but still functional, dystrophin protein.
First, I want to say I'm so glad you are part of PPMD. Your experience, extensive knowledge of DMD and ability to articulate what we as parents need to know about advances in research is extremely valuable.
I am trying to better understand the trials for myostatin inhibition. Is the way that ACE-031 works different from MYO-029 even though both are myostatin inhibitors? I remember eagerly awaiting results of the myodur trials and being so disappointed when wyeth announced it would not continue to study the drug. Is this one substantially different?
Dana, thanks for your note. I agree that Utrophin looks promising and between Project Catalyst results with utrophin at PTC Therapeutics and the Biomarin molecule, there should be at least two clinical trials at some point (would not even hazzard a guess at when). There are also a lot of other ways to upregulate utrophin that are in the preclinical testing stage but look promising. Are you coming to the PPMD Advocacy days on the Hill next week? If so, I'll see you there!
Hi Sharon, You have been flooded with questions. I really just read but I think it's great your trying to help and understand all our thoughts and ideas. My son has a unique deletion 3-36 in-frame. Beckers deletion w/Duchenne progression. I just look out for Utrophin and am praying for a miracle. If I can be of any help in this fight I'm close enough to Washington I will bring my son and put him in front of the world. He is the most beautiful soul God could have gave me. Hope to meet you in Denver. Dana Edwards