Holiday Greetings & Happy New Year 2012
from Coalition Duchenne!
That Was The Year That Was!
2011 has been a year of action for Coalition Duchenne. After its inception in March, Coalition Duchenne, a 501(c)(3) non-profit organization based in Newport Beach, California, held two successful events:
Expedition Mt Kinabalu, Summit of Borneo: On September 9th, a team of 35 international climbers from nine different countries scaled Mt. Kinabalu, the 13,455 ft summit of Malaysian Borneo
Music Power: On November 11th, six incredible bands featured their music to over 600 people at the Galaxy Concert Theater in Orange County, California.
Meeting President Obama
The highlight of the year was a trip in June to Washington DC to privately meet with President Barack Obama in the Oval Office. This was my son Dusty’s wish through the Make A Wish Foundation. We told the President all about Duchenne and Coalition Duchenne. Michelle Obama said of the President that when it comes to the people he has met, he has a memory like a steel trap. He will never forget a story like Dusty’s and that it becomes imprinted on his heart, and he carries it with him every day. Read the story here.
Coalition Duchenne lived up to its mission to raise global awareness for Duchenne muscular dystrophy, and raised much needed funds for research to find treatments and a cure.
Borrowing from Martin Luther King Jr.,
We are confronted with the fierce urgency of now.
Coalition Duchenne has responded.
As the founder of Coalition Duchenne and as a mother of a 19-year-old with Duchenne, there is no time to waste. Even though research has had some major breakthroughs and we are closer than ever before, there is still no cure for Duchenne. At Coalition Duchenne we believe that with more global awareness and with more funding, we will attract more research, treatments and a cure.
Now is the time for humanity to come together to save our sons.
Coalition Duchenne is about acknowledging and bringing together all the world’s Duchenne muscular dystrophy organizations and individuals. But it is more than that. Coalition Duchenne is dedicated to reaching out beyond the Duchenne community and into the world. The world needs to know about Duchenne.
Duchenne is a progressive muscle wasting disease that leaves our son’s minds unaffected and full of dreams. It is the most common lethal childhood genetic disease.
Duchenne affects one in 3,500 boys worldwide.It robs boys of their ability to walk, breathe, eat and speak, and eventually takes their lives, often before they reach age 20. Duchenne affects boys and rarely girls, all over the world, from the USA to Asia, Australia, Europe, India, South America and Africa. It knows no boundaries and does not discriminate between race, culture, socioeconomic status or country.
Moving forward, we are stronger if we all come together, as humans, as a force, as a coalition, bringing everyone together.
Duchenne is a difficult road and presents many challenges along the way. It steals many things, the ability to walk, to hug, to move, and eventually to breathe. But there is that something that it can never get to, that it can never take, and that is hope.
Coalition Duchenne’s logo is a lotus flower, designed by my 12-year-old daughter, Gabriella. A lotus grows out of dark muddy waters, and blossoms into a beautiful flower. The lotus symbolizes enlightenment in spite of our circumstance; it symbolizes our awakening and understanding of the oneness of us all.
Now is the time for humanity to come together. This year, together we climbed the highest peak of South-East Asia and we brought people together to raise awareness for Duchenne through Music Power. Out of diversity, we are one people, connected through love, kindness, compassion and positive energy.
Coalition Duchenne is evaluating promising initiatives in cardiac and pulmonary research. To name just a few: FDA approved drugs such as Sildenafil, and other PDE5 inhibitors, and Spironolactone are potential adjuncts to established ace inhibitors, such as Lisinopril, and beta blockers, such as Carvidelol, to help maintain and even improve cardiac function; Stem cell research is beginning to offer potentially viable treatments although research has been focused on other patient groups; and, several other novel drugs and proteins are close to an FDA investigational new drug application.
Our track record of evaluation and funding is solid. To date, we have raised over $1 million, partly channeling the money through Parent Project Muscular Dystrophy. Independently, we funded the Dusty Brandom Fellowship at the Australian Neuromuscular Research Institute at the University of Western Australia. This helped Dr. Steve Wilton with groundbreaking work on Oligonucleotides (Exon skipping). These nucleic acid polymers are now showing promise in FDA trials. We funded another fellowship with Dr. Andrew Hoey at the University of Queensland working on cardiac research. We have also contributed significant funding to Albuterol (Spencer, UCLA) and small molecules (PPMD’s Project Catalyst). We evaluated the VECTTOR system advocated by Dr. Rhodes in Texas and provided updates to the Duchenne community. Dusty Brandom has also been a long term participant in the Ataluren trial by PTC Therapeutics.
We have worked hard to support boys with Duchenne. We share their achievements with the world through the Our Sons page on our website and advocate for their issues and care. In one example, we worked with a young man in Malaysia struggling with pulmonary issues and hospitalized with pneumonia. We helped his family source a Cough Assist machine and showed him how to use it on Skype. Last year he graduated with a law degree from the University of Malaysia.
We are closely following the latest research initiatives. Web sites such as clinicaltrial.gov are excellent but broad. Treat NMD has synthesized the latest research with links to the specific clinicaltrial.gov number and can be found here. Academic Papers relevant to the Duchenne community published in 2010 and 2011 can be found here.
Coalition Duchenne will be announcing its next investment in Duchenne research early in 2012.
But there is more to do. We continue to respond to the call to action.
Again, with more awareness, we will attract more funding, and treatments and a cure for this disease. This gives new hope and optimism for those living with Duchenne. All money raised in 2011 by Coalition Duchenne will fund cardiac and pulmonary initiatives that will help our sons live longer lives.
All of you are helping to raise awareness and funding to find treatments and a cure for Duchenne.
All of us here at Coalition Duchenne hope that the entire holiday season and New Year will be filled with joy for you and your family.
Please consider making a Christmas or New Year donation to Coalition Duchenne.
Thank you for your support, your kindness and your compassion.
With sincere gratitude,