Please join me and support Cure SMA by making a contribution to my personal fundraising page for the 2016 Philadelphia Rock n Roll half marathon.
I'll keep this short and update you as we approach. Nobody needs any explanation of why I'm doing this, and have done it over the past 4 years. I have many more than just two people I now love that are affected with SMA. I'll be running for my girls, and for my friends and second family out there fighting.
SMA research has moved forward tremendously in the past few years, but the search for a cure is not yet over. With new medications will come new challenges. So this year we #run4ruby #run4emma and #runforSMA.
I’m supporting Cure SMA because spinal muscular atrophy is the number one genetic cause of death for infants, and approximately 1 in every 50 individuals is a genetic carrier for SMA. But there’s great reason for hope. We know what causes SMA and what we need to do to develop effective treatments.
We’re getting closer and closer to an FDA-approved therapy, with 17 ongoing drug programs—and six of those are now in clinical trials.
I’ve chosen Cure SMA because they are uniquely positioned to direct funds to where they can make the greatest difference as quickly as possible. They’ve already invested $57 million in research.
They also provide vital support for families living with SMA, improving their quality of life today. And every year they host the largest SMA conference in the world, bringing families together with the leading SMA researchers.
Making a donation to Cure SMA is easy, secure and completely confidential. Just click on the link and follow the steps!
Thank you again for your personal gift to support my efforts to fund vital research and family support programs for those affected by SMA.
My Personal Web Log
August is SMA awareness month. This is our story.
Last September, shortly after awareness month, a group of friends I hadn't seen in some time came to Philly to support me and my family by running the half marathon. I am continually humbled and overwhelmed by the shows of love and support all of YOU are capable of doing. In truth, it is what sustains me/us. Without these moments when people show me that failure is not an option, I might stumble more often. I literally feel at times like a wobbling wreck and I feel your hands push me up from each side I try to fall. These friends, this gesture and sacrifice, it was more than I could ask, but everything I needed. Until then I had mostly run alone and had made a lot of progress on my abilities in a short time. After this race, I learned how much it could help draw attention to this disease, my girls and possibly help us, this small community of people that care about me and my family, contribute to the therapies we desperately need.
With all of your help, we raised an impressive sum of money through three half marathons, a marathon, and a 10 mile run this past year. My body has never been stronger, and that is also one of the main goals; I will be their legs and arms when they fail them. I will carry the weight.
There is another race unfolding. Its a pharmaceutical race to develop meaningful therapy for SMA. There are 13 companies or researchers in the race. Some have substantial leads. I cheer for all of them. It is complicated. We all want them to succeed, but they need us, our families to succeed. They need us to enroll in trials, and to absorb the risk of trying out their untested therapy to get the drug through the long and arduous FDA process. They need us to subject our kids to pokes and prods, appointments and possible side affects. They need us to say yes to placebo controlled trials - which means some of our kids will be in the trial with all the bad parts of blood draws and electrical tests, while receiving an empty pill. They need this because the FDA requires it to obtain minimal proof of drug safety and efficacy.
I believe the companies truly want to help. Yet I realize they also are in the business of making money off of therapies... First to market drugs will command 200k per child per year in insurance payments. Insurance only has to pay if FDA approves the drugs. So they need us, but we need them. I had the privilege recently of being a family representative at the NIH and FDA meeting called SO-SMART aimed at looking into meaningful outcomes for SMA trials. They are listening to us. I tell others that this is like childhood leukemia in the 70s and HIV in the 80s. We are desperate and will absorb the risk because frankly we know what happens if we don't have a drug. We know that Ruby will deteriorate, and other families with type 1 children will have to say good bye. The FDA and NIH are there to protect us - but I assure you that our small group of family representatives did our best to show them that we are not a passive group. We want fast results. We want a sprint not a marathon.
If the drug companies succeed. Emma and Ruby and all our friends' kids with SMA will be the first generation to have disease modifying therapies. Could this mean that Ruby will walk? Unlikely. It may mean that she continues to use her hands, to text friends, to operate her wheelchair independently. To breathe. So join the stands. Lets cheer on the race and remember that it matters little who wins, as long as they all finish fast.
by Steve Cannady on Fri, Aug 05, 2016 @ 6:40 AM