Most adults with late-onset Pompe disease (LOPD) interviewed after participating in clinical studies of Pombiliti plus Opfolda (cipaglucosidase…
Articles by Andrea Lobo, PhD
High levels of anti-drug antibodies can reduce the availability of Myozyme (alglucosidase alfa) and the amount of the therapy that…
Home infusions of enzyme replacement therapy (ERT) are safe for children with Pompe disease after an initial period…
Children with Pompe disease receiving enzyme replacement therapy (ERT) generally maintained stable breathing during sleep, although some may…
Quantitative MRI (qMRI) may detect muscle changes in people with late-onset Pompe disease (LOPD) before clear declines are seen…
Home-based administration of Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat), an approved combination therapy for late-onset Pompe disease (LOPD), appears…
In the first study of its kind, U.S. researchers showed that focused ultrasound (FUS) could be used to allow approved…
The activity levels of Myozyme (alglucosidase alfa) measured in blood and white blood cells may help indicate how well…
Pompe disease patients bear a substantial burden from coexisting conditions and the ongoing need for supportive care, despite treatment…
Wearable digital health technologies (DHTs) may help identify subtle walking and movement impairments in people with late-onset Pompe disease…