Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results

Abstract Background Olipudase alfa is a recombinant human acid sphingomyelinase (ASM) enzyme replacement therapy (ERT) for non-central-nervous-system manifestations of acid sphingomyelinase deficiency (ASMD). We report 2-year cumulative safety and efficacy data after olipudase alfa treatment in 20 c...

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出版年:	Orphanet Journal of Rare Diseases
主要な著者:	George A. Diaz, Roberto Giugliani, Nathalie Guffon, Simon A. Jones, Eugen Mengel, Maurizio Scarpa, Peter Witters, Abhimanyu Yarramaneni, Jing Li, Nicole M. Armstrong, Yong Kim, Catherine Ortemann-Renon, Monica Kumar
フォーマット:	論文
言語:	英語
出版事項:	BMC 2022-12-01
主題:	Recombinant human acid sphingomyelinase Dose escalation Organomegaly Lung diffusing capacity Acid sphingomyelinase deficiency Niemann–Pick type B
オンライン･アクセス:	https://doi.org/10.1186/s13023-022-02587-0