BioMarin presented five-year data from the PROPEL open-label extension at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan, showing durable motor-function outcomes and relative stabilization of pulmonary function with POMBILITI (cipaglucosidase alfa-atga) plus OPFOLDA (miglustat) in late-onset Pompe disease. The analysis followed 82 adults who continued treatment from the original PROPEL baseline, including 62 participants previously treated with enzyme replacement therapy and 20 who were enzyme-treatment-naïve. Among the ERT-experienced group, the mean prior ERT duration at baseline was 7.6 years. Through year five, the mean change in percent-predicted six-minute walk distance was 0.7%, while the ERT-naïve group recorded a 10.5% change through year 4.5. Percent-predicted forced vital capacity changed by -2.8% and -4.4%, respectively. No new safety signals emerged; 51.2% of participants experienced treatment-related adverse events, and five discontinued for safety reasons, including two because of serious treatment-related events. BioMarin also presented early Phase 1/2 data for investigational antisense oligonucleotide nivudirsen in boys with Duchenne muscular dystrophy amenable to exon 51 skipping, reporting generally favorable tolerability and positive findings across measures including near full-length dystrophin expression.