Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
Küçük Resim Yok
Tarih
2021
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Elsevier
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
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.
Açıklama
Anahtar Kelimeler
Vestronidase alfa, Recombinant human beta-glucuronidase, Enzyme replacement therapy, Mucopolysaccharidosis VII, MPS VII, Phase 1/2
Kaynak
Molecular Genetics and Metabolism Reports
WoS Q Değeri
Q4
Scopus Q Değeri
Q3
Cilt
28
