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A family is trying to raise over $2 million to give their baby a gene therapy treatment to save her life By Alaa Elassar, CNNUpdated 1 hr ago | Posted on Jul 11, 2020 (CNN) -- The family of a 3-month-old girl is trying to raise over $2 million for a one-time gene therapy treatment that may be their daughter's only chance of surviving past her second birthday.On April 1, 2020, at 7:35 p.m., Lucy Van Doormaal was born at a hospital in Vancouver, weighing 7 pounds and 13 ounces, with big bright blue eyes. ... "When we first took her home, we were in newborn bliss. Just absolutely in love with her. She was a great baby," Laura Van Doormaal, Lucy's mom, told CNN. "But we started to realize her movement was declining. She had trouble breathing and eating. When she was five weeks old, she was diagnosed with spinal muscular atrophy."Spinal muscular atrophy, also known as SMA, is a genetic disease that causes infants' muscles to waste away, potentially killing them before age 2. Lucy has type 1 SMA, the most aggressive and severe form of the disease which leaves her nearly unable to crawl, swallow, or even breathe. ...One of the only approved treatments for SMA is Zolgensma, which replaces a working copy of the gene into those nerve cells before they die and symptoms develop. While the US FDA approved treatment is not yet approved in Canada, it is available in multiple countries, including the US.This potentially lifesaving treatment, administered as a one-time infusion into a vein, comes with the hefty price tag of $2.125 million, or around $2.9 million in Canadian dollars. The family is trying to raise $3 million Canadian dollars. ...
