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New 4-Year Data for Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) Presented at ICIEM
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Pombiliti + Opfolda is indicated in the U.S. for the treatment of adult patients with LOPD weighing =40 kg and who are not improving on their current ERT.
In the U.S., Pombiliti + Opfolda is not indicated for patients naïve to ERT treatment.
Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK .
About Late-Onset Pompe DiseaseLate-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA).
Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles that control breathing.
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