Real-world outcomes: LOPD patients who switched to second generation ERT

Real-world outcomes: LOPD patients who switched to second generation ERT

Posted On: 11/06/2025

A chart review was conducted on 15 patients with late-onset Pompe disease followed at a single center who switched from alglucosidase alfa (Lumizyme/Myozyme) to avalglucosidase alfa (Nexviazyme/Nexviadyme) and continued for at least 6 months.

Results: A total of n = 8/15 patients received alglucosidase for more than 3 years prior to switching, and n = 7/15 received it for more than 5 years prior to switching. There were statistically significant improvements in CK, Hex4, and AST

In summary, improved outcomes were seen in most patients, with a subset whose decline persisted. This study presents evidence that switching from alglucosidase to avalglucosidase may be associated with improved outcomes in certain patients with LOPD

For more information please click the link below:

https://pubmed.ncbi.nlm.nih.gov/38313679/