cipaglucosidase alfa-atga (Pombiliti)
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Indications
- in combination with miglustat (Opfolda) for treatment of patients with late-onset Pompe disease*
* adults weighing >= 40 kg not improving on enzyme replacement therapy
Dosage
- 104 mg injection
More general terms
References
- ↑ Meglio M FDA Approves Cipaglucosidase Alfa and Miglustat as First Two-Component Therapy for Pompe Disease. NeurologyLive. Sept 29, 2023
- ↑ Schoser B, Roberts M, Byrne BJ et al. PROPEL Study Group. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. Lancet Neurol. 2021 Dec;20(12):1027-1037. PMID: https://www.ncbi.nlm.nih.gov/pubmed/34800400 Clinical Trial.