Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Molecular Genetics and Metabolism - Tập 116 - Trang 88-97 - 2015
Melissa P. Wasserstein1, Simon A. Jones2, Handrean Soran3, George A. Diaz1, Natalie Lippa1, Beth L. Thurberg4, Kerry Culm-Merdek5, Elias Shamiyeh5, Haig Inguilizian6, Gerald F. Cox7, Ana Cristina Puga7
1Genetics and Genomics Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA
2Manchester Centre for Genomic Medicine, St. Mary’s Hospital, CMFT, University of Manchester, Manchester, UK
3Cardiovascular Trials Unit, Central Manchester University Hospital, Manchester, UK
4Pathology, Genzyme, a Sanofi company, Cambridge, MA, USA
5Clinical and Experimental Pharmacology, Sanofi, Bridgewater, NJ, USA
6Global Safety, Genzyme, a Sanofi company, Cambridge, MA, USA
7Clinical Development, Genzyme, a Sanofi company, Cambridge, MA, USA

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