I had an interesting discussion with Gus today.
“Hey Gus, Mom started climbing today…”
“You understand why she’s doing this, right?”
“Yes, to get money to give to the scientists so they can make me better medicine.”
“If you could get medicine to help you do anything, what would it be?”
“Run…and climb so I could be with Mom right now…”
Yes, they climbed today, their first day on the mountain. All were anxious to get there, but their days in Kathmandu were well spent. Probably the highlight (at least judging from my brief conversations with Tonya) was her visit with Nirmal Khadka and Raju, a 16 year old young man with Duchenne. Nirmal is the founder and chairman of The Muscular Dystrophy Organization of Nepal.
We have known from our work with The Duchenne Alliance that the community of Duchenne patients and their families is worldwide. Tonya felt so honored to experience the strength of that community in her meeting with Raju and Nirmal.
When I think about my conversation today with Gus, I can’t help but think about his Duchenne brother in Nepal who has lived his life in the shadow of the biggest mountain in the world. Unfortunately Raju Khadka is not on the flag that Tonya and team are carrying up Everest. I know, though, that he is in all of their hearts.
Speaking of hearts, once again Gus broke mine today (He does it regularly). Of course he doesn’t understand the complexity of developing medicines. His faith that we’ll ‘make better medicine’ is staggering. It is the challenge we live with daily. I look into Gus’ huge trusting eyes and this week Tonya looked into the eyes of a young man who deserves to climb the mountain he’s lived under his whole life. We know what we have to do.
Specifically, we are considering two very promising trials; one involving gene therapy, another involving a naturally occurring human hormone. Both are potentially ‘better medicine,’ and both are moving to trial. We will get as much expert opinion as we can and we’ll use the money from the Everest team’s climb to fund one of these. We will make sure that every efficiency is in place while maintaining scientific standards of safety and efficacy.
With everyone’s help, Gus’ simple vision will come true. We will provide ‘better medicine’ and we may even see a day where he can run (and climb).
Please donate if you haven’t and thank you!