Lucy Landman, now 3, has PGAP-3 CDG, a rare genetic disorder with severe intellectual challenges, muscle weakness, and seizures, which currently has no treatment.
Gerri Landman emphasizes the agony of watching Lucy suffer daily, noting that her daughter is unlikely to gain independence, speak, or be potty trained.
With both of Lucy's mutations being 'base-editable', there is potential for CRISPR to provide a solution, despite the challenge of funding for rare diseases.
Landman initiated a foundation aimed at finding support and funding for research into single-gene disorders, inspired by a lack of attention from drugmakers.
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