Liver glycogen storage disease type IX (GSD IX) accounts for 25% for all GSD cases, with a prevalence of 1 out of 100,000 patients. GSD IX is caused by deficiency in phosphorylase kinase (PhK), which ...

Glycogen storage diseases (GSDs) constitute a group of inherited metabolic disorders that impair normal glycogen synthesis or degradation, resulting in the accumulation of structurally abnormal ...

CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the ...

PHILADELPHIA (May 16, 2025) – A new study published in Science Advances reveals that a single gene plays a big role in how the liver stores energy, a process that's critical for overall health and for ...

Thirteen-year-old Kayden Soh must follow a strict diet because his liver cannot process sugar. He can only have small amounts of fruit, milk and candy. He also drinks plenty of cornstarch water.

Ultragenyx is gearing up to take its glycogen storage disease (GSD) treatment to regulators next year after the gene therapy helped patients reduce their intake of cornstarch, which is currently used ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...