Comment from Kyrsten Lawless

Summary: Kyrsten Lawless, a parent and advocate for a child with alpha-mannosidosis, argues that the FDA should provide enhanced incentives and streamlined pathways for developing curative AAV gene therapies and pharmacological chaperones. She emphasizes the high clinical and economic burden of current enzyme replacement therapies and advocates for regulatory flexibility to support individualized gene therapies for rare lysosomal storage disorders.