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Cipaglucosidase Alfa: First Approval
Cipaglucosidase alfa (Pombiliti(™)) is a recombinant human acid α-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of t...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10184071/ https://www.ncbi.nlm.nih.gov/pubmed/37184753 http://dx.doi.org/10.1007/s40265-023-01886-5 |