Duchenne is the most lethal childhood genetic disease. In June, families and advocates will join together and shout from mountaintops across the country that DMD needs more attention and more funding for research. CureDuchenne will lead a national publicity campaign for this event and a mini-documentary will record and publicize the efforts of climbers across the country as they fight for their sons.

We are looking for 200 team captains to assemble teams which will each raise a total of $5,000. This means $1 million going directly to research that could potentially save the lives of thousands of boys now and into the future. CureDuchenne will make it really easy to set up your fundraising pages and help you promote your local climb.

Some climbers may desire to attack a major mountain like we did in 2009 when our team of 12 climbed Mt. Rainier. If you would like to target a major mountain, CureDuchenne can help you with the training and logistics. But remember, No mountain is too big or too small.

As one of the 2009 Mt. Rainier team, I can tell you that this was a life changing experience. The camaraderie we developed with our team and the sense of actively doing something that can help our boys was something I’ll never forget.

Please click on these videos to see learn more:

This Year’s 2010 Climb to Cure Duchenne Announcement:

Last Year’s 2009 Mt. Rainier Climb Mini-Documentary:  

Here’s the link to sign up for the climb as a team captain or to find a team that’s being formed. We are happy to answer any questions you have.

Sign Up Today!

See you on the mountain top!

Debra

Debra Miller, Tim Revell, Sally Brown

Parents can make a difference for their boys with Duchenne! 

Tim Revell has been running marathons and hosting golf tournaments in Austin to raise money and awareness for Duchenne muscular dystrophy.  His dedication received the attention of University of Texas head coach, Mack Brown and his wife Sally.  Mack Brown so generously donated to CureDuchenne the $10,000 that he received from the Bobby Dodd Coach of the Year Award.  Sally Brown hosted the “Caffeinate for the Cure” last Friday and has become a spokesperson for CureDuchenne in Austin. 

Channel 8 was there to cover the event:

http://www.news8austin.com/shared/video/video_pop.asp?destlist=65057

 or read about it: 

http://www.news8austin.com/content/sports/?ArID=238219&SecID=5

Tim and his wife Laura had never organized a fund raiser before their son was diagnosed with Duchenne.  They didn’t let that stop them.  I encourage all parents to join in raising money for research…start small and great things can happen.  Our sons are worth the effort it takes and CureDuchenne will support you in getting started.

 Daryl Parrish, father of another Austin boy with Duchenne is running this morning in the Boston Marathon for the CureDuchenne Crusaders.

Bill and Kim Procko also surprised us this week when they announced they will fly out from Florida to attend the  “2009 Dealing for Duchenne” celebrity poker tournament at the Petersen Automotive Museum in Los Angeles.  In addition, Bill and Kim will be bringing with them close to 100 auction items that they have been collecting over this past year.  Chris and Amy Martin, hosts of this event will work with the Procko’s to make this even better than last year’s blockbuster event.

Together we can…Cure Duchenne!

Thanks to the Martin family and Prosum Technology, Title sponsor for Dealing for Duchenne, our first Los Angeles fundraising event was a star-studded success. Held at the famous Petersen Automotive Museum in Los Angeles, this event raised $200,000! Our sincere thanks to Willie Garson (Sex & the City), Anne Heche and James Tupper (Men in Trees), Josh Malina (West Wing), Joel Murray (Still Standing & our celebrity auctioneer), Hayley Norman (Hancock) and Kevin Weisman (Alias) for their compassion and commitment to helping us find a cure for our boys.

Thanks to Ralph Garmon’s promotion of our event on the Kevin and Bean show on KROQ, 450 attendees joined the fun. Everyone had a great time mingling with the stars, dining on fabulous cuisine from Wolfgang Puck’s, gambling and bidding on more than 150 great auction items.

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CureDuchenne gathered a few world renowned experts on Duchenne recently and they were kind enough to talk to us about today’s research and tomorrow’s hope. Please pass this video on to your friends and family. The more the world knows about Duchenne, the sooner we’ll be able to raise the money needed to fund life saving research.

Please be sensitive however, we don’t sugar coat the nature of Duchenne and young eyes should be protected from too much information. CureDuchenne is in the process of evaluating several research projects and we will be excited to share them with you shortly.

Thank you for your continued support,

Debra

Yesterday Prosensa announced commencement of the phase I/II trial for exon skipping at three centers in Europe. This trial will hopefully show safety first, and of course, we are all hoping to see some efficacy as well. Because exon skipping is mutation specific, only a small percentage of Duchenne boys where a skip of exon 51 will restore reading frame will benefit from this particular compound, but other compounds for other deletions are on the drawing board.

Although this is fantastic news we also must remember that this is new technology and still unproved…that is what the trial is for. We really want to thank Dr. Gert-Jan van Ommen from Leiden University and Dr. Judith van Deutekom at Prosensa, and Gerard Platenburg and his team at Prosensa for their dedication to DMD. There are other chemistries in development to induce exon skipping and we will be able to see the benefits of each in the near future.

As we all look to our own sons with hope that a therapy is near, let’s not forget the brave boys that are participating in this trial, they are true pioneers. My prayers are with them for their safety and well being.

On a lighter note, what do Anne Heche (Men in Trees), James Tupper (Men in Trees), Kevin Wiesman (Alias), Willie Garson (Sex & the City), Joel Murray (Still Standing and Bill Murray’s brother), Kevin and Bean (KROQ) and Ralph Garmon (actor and Host of the Kevin & Bean Morning Show) have in common? They are all joining us this Saturday, May 10th at the Petersen Automotive Museum in Los Angeles for “Dealing for Duchenne.” Katrina Carlson will entertain us, Wolfgang Puck will feed us and if you are in the area, please join us. Feel free to pass this on to your friends in LA. A big thanks to Prosum Technology, the titile sponsor for the huge amount of work they’ve done to make this night a success.

-Debra

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