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Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use
Olipudase alfa (Xenpozyme™) is an intravenously administered acid sphingomyelinase enzyme replacement therapy indicated to treat non-CNS manifestations of acid sphingomyelinase deficiency (ASMD) in adult and paediatric patients. It is the first and currently the only disease-modifying treatment for...
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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/PMC10361862/ https://www.ncbi.nlm.nih.gov/pubmed/37133675 http://dx.doi.org/10.1007/s40261-023-01270-x |