Chinese patients with late-onset Pompe disease develop symptoms and are diagnosed at least 10 years earlier than patients from other…
Articles by Patricia Inácio, PhD
Amicus Therapeutics received two U.S. patents for ATB200, a unique recombinant (lab-made) human acid alpha-glucosidase (rhGAA) enzyme, and the…
The first patient has been dosed in a Phase 1/2 clinical trial evaluating the safety and effects of ACTUS-101, a…
Frequent inspiratory muscle training — a set of breathing exercises to strengthen the respiratory muscles making it easier to breathe…
Lower activity of the two lysosomal enzymes underlying Pompe and Fabry diseases — acid alpha-glucosidase enzyme (GAA) and…
Long-term interruption of enzyme replacement therapy (ERT) can lead to negative outcomes for late-onset Pompe disease patients, affecting their lung and exercise…
Newborn screening for lysosomal storage disorders, including Pompe disease, is much more likely to detect individuals at risk for late-onset…
Newborn screening seems better at identifying Pompe disease cases than a clinical examination, especially for classic infantile-onset Pompe disease, according…
Patients with Pompe disease may develop structural changes in the basilar artery — an important artery that supplies oxygen to…
Interim results a from Phase 1/2 clinical trial support the therapeutic benefits of investigational therapy combination ATB200/AT2221 in patients with…