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Molecular Genetics and Metabolism|January 19, 2016
Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durationsTherese Ruane, Mark Haskins, Alphonsus Cheng, et al.Pediatric Research|February 1, 2012
Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI catsDyane Auclair, John Finnie, Steven U Walkley, et al.Clinical and Translational Science|June 2, 2021
Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic diseaseKevin Hammon, Greg de Hart, Brian R Vuillemenot, et al.The Journal of Pharmacology and Experimental Therapeutics|November 19, 2016
Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe DiseaseJeffrey Peng, Jill Dalton, Mark Butt, et al.Investigative Ophthalmology & Visual Science|May 7, 2010
Delivery of SAR 1118 to the retina via ophthalmic drops and its effectiveness in a rat streptozotocin (STZ) model of diabetic retinopathy (DR)Vidhya R Rao, Elizabeth Prescott, Namdev B Shelke, et al.Gene Therapy|February 8, 2022
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapyCarlos Fonck, Cheng Su, Jeremy Arens, et al.Pediatric Research|December 27, 2017
Maternal phenylketonuria syndrome: studies in mice suggest a potential approach to a continuing problemWilliam L Zeile, Helen C McCune, Donald G Musson, et al.Journal of Ocular Pharmacology and Therapeutics : the Official Journal of the Association for Ocular Pharmacology and Therapeutics|March 26, 2010
Safety and pharmacokinetics of a novel lymphocyte function-associated antigen-1 antagonist ophthalmic solution (SAR 1118) in healthy adultsCharles P Semba, Dennis Swearingen, Valerie L Smith, et al.Clinical Therapeutics|December 10, 2014
Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III TrialBecky Schweighardt, Troy Tompkins, Kelly Lau, et al.Clinical Immunology (Orlando, Fla.)|September 12, 2018
Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 studyAnu Cherukuri, Heather Cahan, Greg de Hart, et al.Pageof 5