Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.

Abstract

Vestronidase alfa is an enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII). In this open-label, phase 1/2 study, three subjects with MPS VII received intravenous vestronidase alfa administered every other week (QOW) for 14 weeks (2 mg/kg), followed by 24-week forced-dose titration (1, 4, and 2 mg/kg QOW; 8 weeks each), 36-week continuation (2 mg/kg), and long-term extension (4 mg/kg). Vestronidase alfa was well tolerated and led to dose-responsive, sustained reductions in urinary glycosaminoglycan excretion.

• Authors: Simon Jones, Mahmut Coker, Antonio González-Meneses López, Jennifer Sniadecki, Jill Mayhew, Pauline Hensman, Agnieszka Jurecka
• Journal: Molecular genetics and metabolism reports (Mol Genet Metab Rep) Vol. 28 Pg. 100774 (Sep 2021) ISSN: 2214-4269 [Print] United States
• PMID: 34136357 (Publication Type: Journal Article)
• Copyright: © 2021 Published by Elsevier Inc.