One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

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PURPOSE: To assess olipudase alfa enzyme replacement therapy for non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children. METHODS: This phase 1/2, international, multicenter, open-label trial (ASCEND-Peds/NCT02292654) administered intravenous olipudase alfa e...

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Detalles Bibliográficos
Autores principales: Diaz, George A., Jones, Simon A., Scarpa, Maurizio, Mengel, Karl Eugen, Giugliani, Roberto, Guffon, Nathalie, Batsu, Isabela, Fraser, Patricia A., Li, Jing, Zhang, Qi, Ortemann-Renon, Catherine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354848/
https://www.ncbi.nlm.nih.gov/pubmed/33875845
http://dx.doi.org/10.1038/s41436-021-01156-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354848/
https://www.ncbi.nlm.nih.gov/pubmed/33875845
http://dx.doi.org/10.1038/s41436-021-01156-3

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