long-term

Despite long-term Myozyme, many health problems seen in IOPD kids

Children with classic infantile-onset Pompe disease (IOPD), who were first treated as infants with Myozyme (alglucosidase alfa) — sold as Lumizyme in the U.S. — continue to experience heart, breathing, and cognitive deficits, as well as motor problems, despite long-term use of the older therapy, a small German…

AAN 2023: AT-GAA motor function gains now seen for 4 years in Pompe

Treatment with AT-GAA, Amicus Therapeutics’ experimental two-part therapy for Pompe disease, has now been shown to improve motor function, and to stabilize or improve lung function, for up to four years in adults. That’s according to new analyses from the ongoing Amicus-sponsored Phase 1/2 ATB200-02 trial…

Break in ERT Treatment Linked to Worse Outcomes in Late-Onset Pompe Patients, Study Reports

Long-term interruption of enzyme replacement therapy (ERT) can lead to negative outcomes for late-onset Pompe disease patients, affecting their lung and exercise capacity as well as quality of life, a Swiss retrospective study shows. However, for most patients, resuming ERT can help restore the key clinical parameters that deteriorated during the pause…