A seven-month-old girl with a potentially life-threatening condition has received treatment for free, after millions were raised online for her.
Lucy Van Doormaal was born in April this year with spinal muscular atrophy (SMA), a genetic disease which affects the muscles, causing them to deteriorate over time.
“Quicker than we were even able to finalise her name we noticed something wasn't quite right with our daughter Lucy,” the GoFundMe titled Life for Lucy – Conquering SMA said.
According to the GoFundMe set up by Lucy’s father, Scott Van Doormaal, during the standard newborn check-up, Lucy was observed to have a decrease in both muscle tone and infant reflexes.
The little Canadian girl underwent days of testing and she was then confirmed to have SMA on May 5.
“SMA is a relatively common ‘rare genetic disorder’ that attacks her motor neurons reducing her ability for activities such as sitting, crawling, head and neck control, and swallowing,” the GoFundMe explains.
“Lucy has SMA type I, which is the most aggressive type and left untreated, she wouldn't likely make it to her 2nd birthday.”
The GoFundMe was started to help raise money for treatment, however after more than $3 million was raised for Lucy, her family found out she was a good candidate for a life-saving treatment.
“We just found out that Lucy is a good candidate to receive a groundbreaking gene therapy treatment for SMA called Zolgensma,” her father said in an update on GoFundMe.
Though, getting approved access to the treatment was only half the battle for the family – it is not an approved form of treatment in Canada, where the Van Doormaal’s live, so it would not be covered by health insurance.
All up, the treatment would cost around $3 million, however, in a surprise turn of events, it has been revealed Lucy received the treatment for free.
Lucy was randomly selected by a drug company to receive the treatment for free, CNN reported, and the one time-injection has shown promising results.
Speaking to CNN, Lucy’s mother, Laura Van Doormaal said immediately, she noticed a difference.
"The biggest changes are her head control, really quickly after the treatment she was able to hold her head up which is something she's never done before. She can sit with some support and hopefully one day she'll crawl," Ms Van Doormaal said.
"Maybe one day she'll even walk. That's really an optimistic dream, I know, but we like to have high expectations and we don't want to hold her back."
As the family was able to get the treatment for free, on October 5, they announced people could get a refund if they wanted, though they would be donating money to other children with SMA in Canada.
“One last update about Lucy and the funds - since the original use of the funds has changed, we want to offer the opportunity to ask for a refund if you do not wish for your money to go towards the new plan,” their update on October 5 said.
“We know these donations alone are not enough to get them all Zolgensma right now but we will be able to finish Eva Batista fundraising campaign.
“In addition, donations will be made to Muscular Dystrophy Canada and the B.C. Children’s hospital Foundation to help future SMA children, and lastly some money will be used for Lucy's future and complex medical needs.”
Lucy received the treatment in September this year, though her family is keeping people updated on the GoFundMe and on social media.
1 month post Zolgensma ✨ It’s been just over a month since Lucy got some new genes and what a month of milestones. We are excited that Lucy has developed strength and stamina in her head and trunk control. She can now: -hold her head up when we hold her -sit with assistance ( sometimes floating) -lift her head during inclined tummy time -roll front to back ( on her right side 😅) -move legs against gravity slightly -float like a fish with her otteroo -she smiles a little bit brighter and even started to laugh a little Some of the challenges have been: -weekly blood tests. SMA kids are particularly hard to get blood from and sometimes it’s takes 2 or 3 tries and a bit of fishing. Lucy does not like these ( and neither do I ) - daily steroid dose: Lucy is on a dose of prednisolone to help prevent liver failure. She had to double her dose early on because of elevated liver enzymes levels but have been stable since then. This steroid makes Lucy extra cranky, hungry, restless, nauseous, constipated and sleepless. So overall really great 😅 -but she is taking something to help with the nausea at least. - hand posture. Her thumb seems to collapse in which makes grasping more challenging. She now has little neoprene gloves to wear while playing. They make her look like a tiny boxer which Scott is really into. - the bipap. Because she has been sleeping so poorly, she will not even let us get the bipap mask near her. She has not been wearing it much recently, but her respiratory team would like that to change once she is off the steroid- especially with cold and flu season coming up. Despite these challenges, We have been so encouraged by her progress and excited to see what the next month holds. #nottodaysma #sma1 #lifeforlucy
A post shared by Lucy Van Doormaal (@lifeforlucy_) on
“On Sept 9th, 2020 Lucy received Zolgensma at B.C. Children’s hospital. It was a day charged with emotions as we finally took a deep breath of relief,” the update on September 18 said.
“Our quest for Zolgensma was exhausting yet uplifting. In many ways it changed us as parents, bringing us to extremely dark places but also showing us the amazing strength of human kindness and community.
“We feel so fortunate to have reached our goal of providing this treatment for Lucy. You are all a huge part of this success.”
The ‘Life For Lucy’ Instagram account has over 15,000 followers.
Zolgensma is not a cure for SMA, the GoFundMe says, she will have to live with her condition for the rest of her life, though her parents said they are optimistic.
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