I am hoping to keep you updated on the latest research developments and share whatever information I come across that will help us all take care of our sons in the best way possible.
I am on my home from Washington, D.C., where I was invited to a conference held by Dr. Eric Hoffman at Children’s National Medical Center. Dr. Hoffman and his colleagues have developed CINRG, a clinical trial network for muscular dystrophy that includes 22 clinics in 11 countries. This particular conference was designed to enable clinicians around the globe to prepare for upcoming exon-skipping trials. While we are all hoping and praying that exon-skipping is indeed a viable therapy for DMD, the FDA and regulatory agencies around the world will need to be convinced of its safety and efficacy before our boys will have this treatment available to them.
Scientists came from across the globe to share their latest research on exon-skipping. It was encouraging to see that from a research perspective, we are moving closer to trials and also to see that scientists, doctors and clinicians are thinking ahead in order to have the infrastructure ready for these trials. Thanks are also in order to the Foundation to Eradicate Duchenne, a not-for-profit organization that has provided a great deal of funding for Dr. Hoffman’s lab and DMD research around the world.
I was honored to spend some time with Dr. Shin’ichi Takeda from Japan, Dr. Kate Busby from England and of course, Dr. Hoffman. These busy scientists were kind enough to take a weekend day to help us film an information video for CureDuchenne. We will make this available to anyone who would like to use it for local fundraisers to raise money for DMD research. While this is being edited, please download our PSA (public service announcement) on www.YouTube.com. Feel free to distribute this to friends, family and especially your local TV, radio or newspapers. We all have a responsibility to get the word out about Duchenne.
One question I am asked many times is what supplements are available for our boys. Attached below is our son’s daily regimen. Please keep in mind that these are not prescribed by a physician and no one has established dosage, so do check with your son’s doctor before you use any of these. If you have additional supplements that you think might be beneficial, please write to us and let us know.
Prosum Technology, a Los Angeles corporation, is generously co-sponsoring a CureDuchenne event in Los Angeles on Saturday, May 10th. “Dealing for Duchenne” will be a Texas Hold’em celebrity poker tournament for charity and a casino night. We will enjoy music, dancing, wine tasting, dinner, live and silent auction, while we raise money for Duchenne research. If you live in Los Angeles, please join us. If you know people in the LA area, please pass this registration page to them www.dealing4duchenne.eventbrite.com.
I’ll make no promises as to how often I’ll update this blog, as our small, but growing, staff is pretty busy. Andrea Epstein recently joined CureDuchenne as Executive Director. She has already made a large contribution by streamlining our infrastructure and getting us ready for future growth.
God bless all of our boys,
Debra
PS - If you haven’t yet - be sure to read The Story of CureDuchenne. Click Here
