ask: Treating Muscular Dystrophy using Exon-Skipping Therapy
By
Anonymous
September 3, 2010 - 7:17pm
Rate This
My brothere is 8 years old, he is suffering from duchenne muscular dystrophy, we have been trying to find any cure or treatment since he was 5 years old. We are in desprate need for your help.
Please... Please... we really need your help... and we hope that you might be able to help us in any way!!
Thank you so much! Thank you for your time!
Rate This
All user-generated information on this site is the opinion of its author only and is not a substitute for medical advice or treatment for any medical conditions. Members and guests are responsible for their own posts and the potential consequences of those posts detailed in our Terms of Service.
Add a Comment3 Comments
Dear Anon
Thanks for your post and welcome! Thank you also for advocating for your brother this way and I'm so sorry he's dealing with DMD.
EmpowHER does not conduct clinical trials.
However, I did some research and I think I may have found the trial you are looking for. The first American trial is at Children's Hospital, Columbus, Ohio. However, they were beginning to recruit in June of this year. I don't know if the trial has started yet.
These are the requirements for you brother in order to be suitable for this particular trial:
1.Be diagnosed with Duchenne muscular dystrophy.
2.Have been in a wheelchair full time for at least one year, but no more than four years. If a boy is walking, or uses a wheelchair only part time, he will not be a candidate for this study.
3.Have one of the following out-of-frame deletions in the DMD gene:
•exons 13-50
•exons 29-50
•exons 43-50
•exons 45-50
•exons 47-50
•exons 48-50
•exons 49-50
•exon 50
•exon 52
You can read more here, as well as find contact information: http://quest.mda.org/news/first-us-exon-skipping-trial-opens
You can also contact the Muscular Dystropy Association to find more about upcoming clinical trials or for a local contact.
www.mda.org
Also, please head to our MD page where you can read more, as well as informative articles and information that can help you further.
http://www.empowher.com/condition/muscular-dystrophy
Please let us know if this helps and we wish you and your brother the very best,
September 4, 2010 - 1:02pm~Susan
This Comment
Dear Susan
Thank you for your help! I really appreciate it!
September 4, 2010 - 4:34pmWe will be contacting the Study Coordinator: Xiomara Quintero Rosales, M.D.
Thank you again and I would like you to inform me of any new treatments concerning DMD.
Thank you
Anon - EmpowHER has more than one million visitors every month and we are not able to provide updates to individuals. The best way to stay informed is to register as a member of the site and to stay in touch. We have information that will help your entire family take care of their health.
September 6, 2010 - 1:32pmBest wishes for your brother. Please keep us posted on his progress.
Pat