In Pompe disease, mutations in the enzyme responsible ... 71 (CD71), a protein found on the surface of muscle cells. Once inside the muscle cells, the siRNA molecule can then act on glycogen ...

which is deficient in Pompe disease, leading to the accumulation of glycogen to toxic levels that damage muscle cells. Disease severity ranges on a spectrum, but predominant manifestations are ...

The drug is a recombinant version of the enzyme alpha-glucosidase, which is deficient in Pompe disease, leading to the accumulation of glycogen to toxic levels that damage muscle cells.

Dublin, Nov. 21, 2024 (GLOBE NEWSWIRE) -- The "Pompe Disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering. The report provides comprehensive ...

“The limitations of enzyme replacement therapy for Pompe disease underscore the need for new therapies with novel mechanisms of action,” said Aneal Khan, MD, principal investigator of the ...

Dublin, Nov. 21, 2024 (GLOBE NEWSWIRE) -- The "Pompe Disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering. The report provides comprehensive insights ...