Patient-derived Nerve Cells May Help Advance Pompe Research

As a proof-of-concept, the team evaluated the effects of treating the cells with a lab-made GAA — obtained after infusions of Lumyzime (alglucosidase alfa), an enzyme replacement therapy — delta-tocopherol, and hydroxypropyl-beta-cyclodextrin (HPβCD). Delta-tocopherol and HPβCD are two structurally distinct small molecules previously shown to lessen disease features in cellular and animal models of lysosomal storage diseases.

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