My son has Duchenne muscular dystrophy. I am a software developer and a father, and this page exists because I cannot do this part on my own.
Duchenne is caused by an error in the gene that makes dystrophin, the protein muscles need to repair themselves after ordinary use. Without enough of it, muscle is slowly lost and replaced. In my son's case the error is a pathogenic point mutation in exon 70: a single misspelled letter, in one section of one gene, out of everything that makes him who he is.
It moves faster than people expect. He can no longer climb more than one stair. Muscle that is lost does not come back, so every month that passes before treatment is a month that cannot be recovered.
There is a one-time gene therapy for Duchenne called Elevidys. It delivers a shortened working copy of the dystrophin gene into muscle, so the body can make a version of the protein it cannot currently produce.
It is not a cure, and I will not pretend otherwise. It is not expected to undo what has already happened. What his doctors hope is that it slows down what comes next, and buys him years of walking, of climbing stairs on his own, of being a boy rather than a patient.
The therapy carries a list price of approximately US$3.2 million for a single dose. That is not a number a family reaches by saving.
Write to me and I will tell you exactly where things stand and what is needed. I would rather have that conversation than put a collection tin on a page.
Write to meShare this page. Reaching people is most of what a fundraiser is. One person who knows a hundred others is worth more than a small donation.
Play the games and tell someone. I make small games, and what they earn goes to the same place. Wake the Garden is free on the App Store. It will not raise three million dollars, and I know that. It is simply the part I can do myself instead of only waiting.
Treatment decisions, eligibility and timing rest entirely with his medical team. Nothing on this page is medical advice, and I speak only for my own family.
Thank you for reading this far.