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

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...

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Detalles Bibliográficos
Autores principales: Jones, Simon, Coker, Mahmut, López, Antonio González-Meneses, Sniadecki, Jennifer, Mayhew, Jill, Hensman, Pauline, Jurecka, Agnieszka
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178115/
https://www.ncbi.nlm.nih.gov/pubmed/34136357
http://dx.doi.org/10.1016/j.ymgmr.2021.100774
Descripción
Sumario: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.

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