Williams said the disease was the result of a mental health struggle. “With body dysmorphia, when people say they’re worried about how you’re looking, you’re like: ‘I’ve achieved it ...

which remains the only other FDA-approved therapies for Pompe. The company estimates there are around 3,500 people in the US with both early- and late-onset forms of the disease. The FDA approval ...

Shionogi has concluded an exclusive global licence agreement for Maze Therapeutics’ MZE001, an oral glycogen synthase 1 (GYS1) inhibitor to treat Pompe disease. The agreement grants Shionogi the ...

Pompe disease is characterized by a deficiency of acid α-glucosidase (GAA). The GAA enzyme degrades lysosomal glycogen, and insufficient GAA activity causes glycogen to accumulate in various tissues.