New 4-Year Data for Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) Presented at ICIEM
1. New long-term data confirms efficacy of Pombiliti + Opfolda in Pompe disease. 2. Improvements in muscle function maintained for 208 weeks in ERT-experienced patients. 3. Study involved 82 patients; no new safety signals were identified. 4. Upcoming pulmonary function data will further inform treatment profile. 5. Pombiliti + Opfolda is not indicated for treatment-naïve patients.