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Durable five-year data for LOPD support long-term value of POMBILITI + OPFOLDA, appealing to biotech investors and potentially lifting sentiment and multiple on BioMarin's rare-disease portfolio; limited near-term catalysts but strengthens pipeline credibility.
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BioMarin reported five-year PROPEL open-label extension results for POMBILITI + OPFOLDA in late-onset Pompe disease, showing durable motor function and stabilized pulmonary decline with manageable safety. The gains were larger in previously untreated patients, while ERT-experienced users demonstrated smaller but positive improvements; data were presented at the World Muscle Society Congress in Hiroshima. These long-term findings support the therapy's value proposition and could bolster investor sentiment around BioMarin's rare-disease portfolio, particularly if ongoing follow-ups continue to confirm durability.
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