When their baby Lucy was five weeks old, parents Scott and Laura Van Doormaal learned that she had a potentially fatal condition called spinal muscular atrophy (SMA). The gene therapy treatment Lucy needed to save her life was expensive, costing over $2 million, and the family worked hard to raise the funds. Then a miracle happened. Lucy was selected by drug maker Novartis for the Managed Access Program, which gave her access to the treatment for free. Lucy has received the treatment and is showing signs of improvement; her family is hopeful for her future. The funds raised for Lucy’s treatment have been donated to other babies with SMA who need the same treatment and a few charities, and some will support Lucy’s future medical needs.

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