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This ongoing, open\u2010label, long\u2010term study (NCT02004704) assessed safety and efficacy of olipudase alfa following 30 months of treatment in five adult patients with ASMD. There were no deaths, serious or severe events, or discontinuations during 30 months of treatment. The majority of adverse events were mild and included headache, nausea, and abdominal pain. No patient developed anti\u2010drug antibodies and there were no clinically significant adverse changes in vital signs, hematology, or cardiac safety parameters. Statistically significant reductions in liver (31%) and spleen (39%) volumes were maintained through 30 months of treatment. There was a mean increase in lung diffusing capacity of 35%, and clinically relevant improvements in infiltrative lung disease parameters. Lipid profiles improved in all patients. Improvements in bone mineral density of the spine were observed in some patients. Chitotriosidase in serum and lyso\u2010sphingomyelin in dried blood spots decreased with olipudase alfa treatment, suggesting utility as biomarkers for monitoring treatment efficacy. Olipudase alfa is the first etiology\u2010specific treatment in development for ASMD. This study demonstrates that treatment with olipudase alfa for 30 months is well\u2010tolerated and associated with life\u2010transforming sustained improvements in relevant disease clinical measures.<\/jats:p>","DOI":"10.1007\/s10545-017-0123-6","type":"journal-article","created":{"date-parts":[[2018,1,5]],"date-time":"2018-01-05T05:01:00Z","timestamp":1515128460000},"page":"829-838","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":108,"title":["Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months"],"prefix":"10.1002","volume":"41","author":[{"given":"Melissa P.","family":"Wasserstein","sequence":"first","affiliation":[{"name":"Children's Hospital at Montefiore Albert Einstein College of Medicine Bronx NY USA"},{"name":"Genetics and Genomics Sciences Icahn School of Medicine at Mount Sinai New York NY 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