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.

“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 ...

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 ...