Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
Makaleler > Tam Makale · 2021
Yazarlar: Simon Jones, Mahmut ÇOKER, Antonio Gonzalez-Meneses Lopez, Jennifer Sniadecki, Jill Mayhew, Pauline Hensman, Agnieszka Jurecka
MOLECULAR GENETICS AND METABOLISM REPORTS, SCI Expanded İndekslerine Giren Dergi, Uluslararası, Scopus, 2021, cilt 28, ss 100774-100774
DOI: 10.1016/j.ymgmr.2021.100774
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.