| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8343766 | Molecular Genetics and Metabolism | 2015 | 10 Pages |
Abstract
This study provides proof-of-concept for the safety and efficacy of within-patient dose escalation of olipudase alfa in patients with nonneuronopathic ASMD.
Keywords
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Authors
Melissa P. Wasserstein, Simon A. Jones, Handrean Soran, George A. Diaz, Natalie Lippa, Beth L. Thurberg, Kerry Culm-Merdek, Elias Shamiyeh, Haig Inguilizian, Gerald F. Cox, Ana Cristina Puga,