Devin and I were at the University of Florida for the Imaging DMD study. Devin was in a room going through a series of functional tests to assess his muscle strength, while I sat in a waiting area doing some work on my computer. I noticed a lady and her son walking into the same room I was in when, all of a sudden, she asked if I have a son with Duchenne. I was a little puzzled, but then I realized I was wearing my CureDuchenne shirt. It’s pretty cool knowing we can raise awareness in such a simple way. Anyway, I told her that I do in fact have a son with DMD and that I was sitting there waiting for him.
I thought that would be the extent of the conversation, but then she asked me if I knew what happened to the drug that was shown to be effective, but not available to everyone. She went on to say that she read about it a few years ago in The Economist (I couldn’t find that article, but linked to one that’s similar). I wasn’t sure which drug she was referring to, but I had a few ideas. Then she said the article was about a mother with two boys who have DMD and one of the kids was able to get the drug, but the other son was not. That was enough information for me. I knew who she was referring to and which drug she was wondering about.
“It’s called eteplirsen. The FDA approved that drug last year. My son is actually taking the medicine,” I said. Then I told her both boys in the article she read now have access to the medicine. Her eyes got really big and the look on her face expressed pure joy. It was unlike anything I expected. The lady said she had been praying for the family since the day she read the article a few years ago and was so happy to hear such wonderful news.
Whoa! I thought, this lady has been praying every day for years for someone she didn’t even know based on an article she read about their situation? I honestly couldn’t wrap my head around that. I mean, I’ve prayed for people I didn’t know, but not like that. I’d say a prayer or maybe pray each day for a week, but I have never prayed for anyone I didn’t know every day for years! I didn’t know how to react except to thank her as best I could. I told her I know the family and I asked if I could relay a message to her. The lady stood up, walked over to me, and gave me a hug! As if I wasn’t already shocked, nowwwww my mind was totally blown!
Her son began to need more of her attention at this point, but she mentioned they are involved in a situation that is somewhat similar. Her and her son were there for a clinical trial. He has Lennox-Gastaut syndrome, which is a form of epilepsy. It produces severe seizures that have caused his heart to stop on multiple occasions. She said he’s coded more than once. He used to experience anywhere between 10-50 seizures each day, but it’s been a lot better since he began taking the investigational medicine known as epidiolex. In fact, he only had eight seizures through the entire month of March and only three in April (as of 4/25). She was called into her son’s appointment just then, but not before I could tell her that I will be praying for her, her son, and everyone else who may benefit from epidiolex. With that she left me saying, “God bless you.”
It was abundantly clear, He already has.