Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency.

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Published in Am J Surg Pathol on September 01, 2016

Authors

Beth L Thurberg1, Melissa P Wasserstein, Simon A Jones, Thomas D Schiano, Gerald F Cox, Ana Cristina Puga

Author Affiliations

1: Departments of *Pathology †Clinical Development, Sanofi Genzyme, Cambridge ¶Division of Genetics, Children's Hospital Boston #Department of Pediatrics, Harvard Medical School, Boston, MA ‡Division of Pediatric Genetic Medicine, The Children's Hospital at Montefiore, Bronx ∥Division of Liver Diseases, Mount Sinai School of Medicine, NY, NY §Manchester Centre for Genomic Medicine, St Mary's Hospital, CMFT, University of Manchester, Manchester, UK.

Associated clinical trials:

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients | NCT01722526

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