After three months of fundraising on the GoFundMe platform, Scott Van Doormaal and Laura Kendall’s dreams were realized when their baby daughter Lucy was selected in a lottery system to receive the lifesaving drug she needed.
Lucy was born April 1, and was diagnosed with type 1 spinal muscular atrophy (SMA) just weeks after her birth. Since her diagnosis, her parents were desperate to find a way to help their baby girl, and in May they started a fundraiser to ask the community to help them raise the $3 million needed for the treatment.
After a few months they nearly hit their goal, raising more than $2.4 million towards the purchase of the drug Zolgensma. In August, Lucy was selected in a lottery to receive the drug, and on September 9 she had the drug administered.
“All those kids, when they are diagnosed and approved for treatment, are entered into what’s called a managed access program put on by the drug company. And it essentially acts as a lottery. So it’s a worldwide lottery system, and every two weeks, they select a child to receive the treatment. And to our absolute shock, Lucy was selected in August,” said Kendall, who’s originally from Elmira. “Because it’s a degenerative neuromuscular disease, time is so important. So, every day was critical. And this allowed us to get the treatment sooner, so that was amazing.
“It’s very hard to describe the experience. Honestly, I think that being handed such a terrible diagnosis was really, really tough for our family and for our community as well. But having to put your life out there and ask so many people [for help] and fundraise for her life, I can only describe it as like a traumatic experience.”
Getting Lucy prepared for the treatment was a long road. The infant went through blood, physio and other tests to ensure that she was still able to make the list to get the treatment, eventually receiving it last month.
Since getting her treatment, Lucy has been getting better and while there have been ups and downs, they have seen many significant improvements in her life.
“Her liver enzymes [spiked] on week two, and we had to increase her dose of steroids to make sure that she was safe and healthy. But since then, she’s been doing fairly well on it. And hopefully, she’ll be able to wean off the steroids soon,” said Kendall.
“Physically, we noticed a difference right away; within a few days, she started to hold her head up, which is not something she could do before the treatment, she started to move her legs a little bit more. And she started to have a little bit more strength and endurance in her core. So what she’s been able to do with all of those progression is she has now lifted her head for the first time, which is not that big of a deal in terms of babies, because … one-month old babies can do that. But that’s something that she’s never even shown that she might be capable of doing – that’s really encouraging.”
Lucy has started to practice sitting, and can now sit for about five seconds by herself, Kendall added.
“That’s not something SMA type 1 babies typically are ever able to do. So it’s pretty miraculous what we’re seeing. She’s got a really long road ahead, and we do have to complement the treatment with a lot of physiotherapy. We see an occupational therapist, and we see a feeding therapist,” she said. “Honestly, within the first month I don’t know that we were expecting such a drastic change right away.”
Now that Lucy has received her treatment, the money raised by the family is now going to help other babies with SMA.
Kendall says there are seven babies that are going to be helped by the funds raised, including paying the additional cost needed for one to receive the needed Zolgensma treatment. Aside from paying to help other kids, money will go to a local hospital and other funds, in addition to saving money for Lucy’s future.
Kendall extended a thank-you the community for the work they did to help Lucy.
“It’s really hard to articulate how impactful that was, especially because we don’t live in that community anymore. But we visit frequently, obviously, because our family lives there. This has been a really hard experience for us, obviously. But it’s been a hard experience for our family. And the community supporting our family, I know has made a huge difference,” she said. “It was so overwhelmingly positive, to receive that magnitude of support and the passion that everybody had to get Lucy, the best treatment possible, and to help support our family was amazing. And I just think it speaks magnitude to the close knit community and it actually made me kind of miss being there a little bit. But, obviously, people make the place so much. We’re very grateful.”