"Faith is the very first thing you should pack in a hope chest." ---- Sarah Ban Breathnach
As 2011 begins, I am hopeful that there will be new research breakthroughs for Duchenne. My family is grateful to the generosity of the Getler and Ginder families in support of PPMD’s cardiac initiative. We begin every year with a bitter sweet celebration. New Year’s Eve of 1997 began our journey. It’s been 14 years since Duchenne entered our lives. Jon has always told me that Duchenne does not define him, it’s just something that he deals with. He has never let Duchenne stop him from trying something he was interested in.
2010 celebrated normal high school milestones, prom, participating in Model UN conferences, SAT’s, ACT’s. 2011 finds us looking forward to new milestones, picking colleges Senior Ball, Senior Bash and Graduation.
Jon is always hopeful for new research breakthroughs. Even though traveling from Western NY to Utah causes some stress and extra work at school, Jon is thankful that he is able to be back on Ataluren. As we were driving the other day, Jon said “Mom, I feel pretty fortunate that I’m able to participate in the Ataluren trial, cause I’m still mobile. Will the FDA allow trials for the non-ambulatory kids, I think it’s unfair that kids who can’t walk, can’t participate in trials for new therapies.” “Can’t they figure out some kind of measurements so non-ambulatory kids could participate in these trials?” I responded with: “I know that they are working on it, but it’s not easy. Drug companies need to show patient benefit for a drug. They need to show a measureable benefit that will provide a medical qualitative benefit.” Jon’s response was “Mom, that’s pretty complicated to figure out, I hope they will figure it out soon, I will always remain hopeful and have faith that in my lifetime we will see that happen as well.”
Jon and I end 2010 and begin 2011 with faith and hope that research breakthroughs will help END Duchenne.