A Family That Raised $2 Million for Their Baby's Life-saving Medical Treatment Has Received It for Free
Oct 25, 2020
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A Canadian family that spent months frantically raising millions of dollars for a one-time gene therapy treatment to save their daughter's life has received the treatment for free.
Lucy Van Doormaal, now 7 months old, was born with spinal muscular atrophy (SMA), a genetic disease that causes infants' muscles to waste away, potentially killing them before age 2.
After her family raised nearly $2 million to pay for a potentially life saving treatment, administered as a one-time infusion into a vein, Lucy was randomly selected by drug company Novartis Gene Therapies to receive the treatment for free through its Managed Access Program (MAP).
"We were totally shocked, we didn't expect this outcome but we were just so happy because our single goal was providing the treatment to Lucy," Laura Van Doormaal, Lucy's mom, told CNN. "This has been a traumatic experience, having to fight for treatment and put our life out there to fund it, so this is really a relief."