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: ...

Discover the top benefits of Lion's Mane mushroom for health. Learn how this powerful fungus can boost brain function, ...

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 ...

The thing is that Pombiliti + Opfolda were approved by the FDA for the treatment of adults with late-onset Pompe Disease weighing ≥40 kg and who are not improving on their current enzyme ...

A new generation has emerged: Generation Alpha. While the official age range for these youngsters is still heavily up for debate (some maintain the generation began in 2012 and runs through 2024 ...

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 ...

While there’s no release date for the game quite yet, you can still test it out early to see if Destiny: Rising can live up to Bungie’s standards by applying for the upcoming alpha test.