9,828 miles
That’s approximately the cumulative, round-trip distance from our home to a gene therapy clinical trial site which, with a few more visits still ahead, we will have visited 14 times this summer. The way I feel about this opportunity, to provide Charlie with the best relatively proven option science currently has to offer him, can be summed up this way: I feel I can exhale for the first time since diagnosis. The initial rush of nonstop adrenaline has relaxed into what I would assume is a normal sense of urgency. Everyone knows this drug is not a cure. But it’s a start. It is maybe putting the runaway train of Duchenne intro neutral for a few years until scientists can send us something else. And that sense of relief is everything.
We are indescribably grateful to have qualified for this trial, to have the means to make these trips happen—no doubt about that. We are also exhausted. While we watched other families jaunt to Europe or play nonstop in sand and surf, we logged hours on interstates and in hotel rooms. We pinned Charlie down week after week with three nurses (he is ironically and freakishly strong) in order to draw his requisite labs. Our immune systems latent from Covid cautiousness and perhaps a little weakened by fatigue, 3/4 of us came down with some evil virus during what was supposed to be our one, blissful week of a trial trip hiatus and true family vacation. At the end of that same trip, after driving to the trial site, we got a flat tire on the drive home. It began to feel like we should light some sage.
I am not complaining; we would make these same choices and trips a million times over and then some. Science is hard work, but it is worth every obstacle, every strange summer, to have this dose of hope. To be able to help potentially advance this therapy toward approval and share this hope with the full DMD community. I can’t help but think of all the families who came before us, who logged miles on long stretches of highway and in airports across the globe to get us to this point; I gratefully salute you. And I stand with all the mothers, fathers, siblings and grandparents whose every hope hangs on this treatment in trial, and all the discoveries still to come.