For adults with late-onset Pompe disease (LOPD), Pombiliti + Opfolda (cipaglucosidase alfa/miglustat) appears to offer lasting benefits. New…
Articles by Lindsey Shapiro, PhD
A genomic medicine designed to correct the genetic defect found in up to 90% of people with late-onset Pompe…
Children with infantile-onset Pompe disease (IOPD) experienced reductions in disease-related biomarkers and stabilizations or improvements in clinical function after…
The combination therapy Pombiliti + Opfolda (cipaglucosidase alfa/miglustat)Â may help overcome some of the limitations of standard enzyme replacement…
A woman in her 30s with severe respiratory failure and muscle weakness due to late-onset Pompe disease (LOPD) repeatedly…
A therapeutic approach designed to correct the cellular defect caused by a common mutation in people with late-onset Pompe…
Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) may offer significant advantages over earlier forms of enzyme replacement therapy (ERT) for Pompe…
Most children and adolescents with Pompe disease adapted well to living with the chronic condition and had generally good…
A new noninvasive imaging technique was found to be better than standard approaches like MRI or ultrasound for visualizing diseased…
Damage to the blood vessels in the brain is a common but under-recognized manifestation of late-onset Pompe disease (LOPD),…