Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice
Allyson L. Anding; Sofia Kinton; Kaitlyn Baranowski; Alexander Brezzani; H. de Busser; Michael R. Dufault; Patrick F. Finn; Kelly Keefe · 2023 · Journal of Pharmacology and Experimental Therapeutics
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract (excerpt)
Pompe disease is a rare glycogen storage disorder caused by a deficiency in the lysosomal enzyme acid α-glucosidase, which leads to muscle weakness, cardiac and respiratory failure, and early mortality. Alglucosidase alfa, a recombinant…
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Metadata source: OpenAlex · DOI 10.1124/jpet.123.001593