Lucy may not live to see her second birthday without expensive gene therapy

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baby lucy
Credit: Scott and Laura Van Doormaal

Lucy Van Doormaal was born on April 1 in Vancouver, but before her parents, Scott and Laura Van Doormaal, could “finalize her name,” they noticed that “something wasn’t quite right” with their daughter, according to their GoFundMe page.

A month later, Lucy was diagnosed with Spinal Muscular Atrophy (SMA), a rare genetic disorder that attacks motor neurons and weakens the voluntary muscles used for crawling, walking, head and neck control and swallowing.

Because Lucy’s particular case of SMA is Type I, which is the most aggressive type, her parents explained on the fundraiser that their daughter likely wouldn’t make it to her second birthday.

With little to no other options, the Van Doormaals are now hoping that Lucy’s GoFundMe page will help them raise enough money to afford a “groundbreaking gene therapy treatment” called Zolgensma, which could ultimately save their daughter’s life.

Related article:  Animal safety concerns prompt FDA to halt study testing Zolgensma gene therapy for spinal muscular atrophy
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Created on May 10, the GoFundMe officially hit the $1 million mark on [July 11] with Scott thanking the 38,800 donors — including members of the Hollywood community, such as Ryan ReynoldsOlivia Wilde and Joshua Jackson — for their support.

“It’s unbelievable the amount of support we have been getting from all over the globe. We want to take a moment and make sure that we truly pass along our gratitude to each and every donor,” Scott wrote. “Together we are making this happen for Lucy.”

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