BioMarin Reports Five-Year Stability in Pompe Disease Treatment

The findings from the PROPEL open-label extension study tracked 82 individuals who received the combined treatment continuously. Participants included both those previously experienced with enzyme replacement therapy and treatment-naïve patients. For the cohort with prior therapy experience, the mean duration of previous treatment stood at 7.6 years before enrolling in the study. Clinical outcomes showed relative stabilization of pulmonary function and durable motor function, supporting the long-term viability of the drug combination in managing this progressive genetic condition.

Safety profiles remained consistent with previous observations, and no new signals emerged during the five-year follow-up. While 51.2% of participants reported treatment-related adverse events, most were described as manageable. BioMarin also highlighted progress on nivudirsen, an investigational treatment for Duchenne muscular dystrophy, which demonstrated positive dystrophin expression and favorable tolerability in early-phase trials.