11.3. The Advisory Committee recommended that avalglucosidase alfa for the treatment of late onset Pompe disease be funded with a medium priority.

Q3 2024 Total Revenue of $141.5M, a 37% Increase Year-over-Year Galafold®Q3 Revenue of $120.4M, up 20% Year-over-Year PRINCETON, N.J., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: ...

The reason why this disease occurs is because of a mutation in the GAA gene, which is very bad considering that it is responsible for producing the alpha-glucosidase enzyme. This particular enzyme ...

as they are carried by the flows produced by a β-glucosidase micropump in the presence of 20 mM cellobiose (AVI) Video of tracer particles, from an observation plane 170 μm above the glass wall with ...

To determine the requirement of glycogenolysis for migration or survival in extracellular matrix (ECM) detached conditions, siRNA inhibition of glycogenolysis (liver glycogen phosphorylase, PYGL) or ...