PATTI: My daughter Adalyne, Addy, has SMA Type 1. Our first daughter Madison was born back in 2004 and at that time we knew nothing about SMA. Unfortunately, the disease really was tough on her body and at the age of nine months, she was G-tube fed and by the time she passed at 3½, she was unable to move any part of her body except for her finger. Maybe because I was a little paranoid, I was watching early on. With Addy, I really started noticing a lot of the same symptoms that Madison had.
BRANDON: She had considerable head lag. You could tell that her, you know, her extremities were definitely getting weaker. It was almost instantaneous where she was able to quit moving her legs.
PATTI: We decided to go to the doctor with Addy around six weeks of age. We went and had her tested. Our pediatrician really felt like it was gonna come back negative. It took about a week. She just happened to be working the next day on a Saturday and happened to tell her nurse to go check and see if anything had come in, not expecting that it had, and it had come in. And she said she was quite devastated herself.
BRANDON: Addy was diagnosed in September of 2015.
PATTI: Our pediatrician knew that we were pretty advanced in knowing people who knew what’s going on out in the world, and the first thing she said is “start doing your research.”
BRANDON: I found several different clinical trials that were recruiting patients. And we came upon ZOLGENSMA and, you know, I made one phone call, and they called us back that, that very night, and said “we’d like to see your child.”
PATTI: They took a working gene and sent it to replace the nonworking gene by doing an infusion that took roughly an hour. I will always remember the day when Addy received her treatment. It was at 3:42 on October 28th. I remember telling my husband, I feel like she’s having another birthday. The biggest thing that we noticed was her head control. She was able to sit unassisted for a very lengthy time.
BRANDON: You see her starting to be able to move her legs more, move her feet more, move her head. ZOLGENSMA was able to give Addy a second chance to be able to breathe freely to be able to roll herself around freely. She continues to get stronger little by little every day. To the outside eye, it may not be anything. But when you live with her every day, you see these little small things.
PATTI: We do a lot of therapy with her. She just aged out of early intervention, so prior to aging out, we did occupational and physical therapy at home as well as off site. We do water therapy with her. Again, this treatment has changed her life tremendously, but we still have a lot of underlying issues that we have to deal with that come along with having a child in a wheelchair. It’s important for people to understand that regardless of the treatment, your child has this diagnosis that’s gonna follow them for the rest of their life. Stopping the progression of SMA means not watching my child decline. It means watching her actually have the ability to feed herself. Have the ability to propel herself in her wheelchair, which is something I never thought I would see. Parents should know about ZOLGENSMA because having something that they receive one time changed our life tremendously. It’s changed Addy’s life tremendously.
Close the transcript