A Canadian family who spent months trying to raise millions of dollars for a life-saving treatment for their daughter has received it for free. Lucy Van Doormaal, was born with spinal muscular atrophy or SMA, a genetic disease that causes infants’ muscles to weaken and potentially kill them by age two. Her family raised nearly $2-million for the one-time gene therapy treatment, but the baby girl was randomly selected by drug company Novartis Gene Therapies to get it for free from its Managed Access Program.
The treatment has a sky high price tag of $2.125-million and alters the patient’s biology, but it’s still not guaranteed to be a cure. But getting it was Lucy’s only chance of surviving, so her family was frantically trying to come up with the money. And now that she’s had it? The seven-month-old is already showing significant improvement.
“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,” her mom, Laura explains.
The money that the family raised has been donated to other babies with SMA who need this treatment, along with some charities, and the rest of the money will go to Lucy’s future medical needs.
Source:CNN