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Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months.
Wasserstein, Melissa P; Diaz, George A; Lachmann, Robin H; Jouvin, Marie-Hélène; Nandy, Indrani; Ji, Allena J; Puga, Ana Cristina.
Affiliation
  • Wasserstein MP; Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, USA.
  • Diaz GA; Genetics and Genomics Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Lachmann RH; Genetics and Genomics Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Jouvin MH; National Hospital for Neurology and Neurosurgery, London, UK.
  • Nandy I; Agios Pharmaceuticals, Cambridge, MA, USA.
  • Ji AJ; Clinical Sciences and Operations, Sanofi Genzyme, Cambridge, MA, USA.
  • Puga AC; Clinical Sciences and Operations, Sanofi Genzyme, Cambridge, MA, USA.
J Inherit Metab Dis ; 41(5): 829-838, 2018 09.
Article in En | MEDLINE | ID: mdl-29305734
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sphingomyelin Phosphodiesterase / Recombinant Proteins / Niemann-Pick Disease, Type A Limits: Adult / Female / Humans / Male Language: En Journal: J Inherit Metab Dis Year: 2018 Document type: Article Affiliation country: United States Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Sphingomyelin Phosphodiesterase / Recombinant Proteins / Niemann-Pick Disease, Type A Limits: Adult / Female / Humans / Male Language: En Journal: J Inherit Metab Dis Year: 2018 Document type: Article Affiliation country: United States Country of publication: United States