vestronidase alfa

09/01/2021 - "Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII."
09/01/2019 - "Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population."
01/01/2018 - "To overcome these challenges, a novel Blind Start design was utilized in a study of vestronidase alfa in mucopolysaccharidosis VII (Sly syndrome), an ultra-rare lysosomal disease, that demonstrates the strengths of this approach in a challenging drug-development setting. "
04/01/2019 - "Given the lack of treatment options and the clinical benefits it provides, intravenous vestronidase alfa is an important emerging ERT for patients with mucopolysaccharidosis VII."
04/01/2019 - "Vestronidase Alfa: A Review in Mucopolysaccharidosis VII."

03/09/2023 - "Enzyme replacement therapy (ERT) for MPS VII using recombinant human β-glucuronidase (vestronidase alfa) was recently approved for use in patients; however, to date there have been no studies evaluating therapeutic efficacy in a large animal model of MPS VII. The objective of this study was to establish the effects of intravenous ERT, administered at either the standard clinical dose (4 mg/kg) or a high dose (20 mg/kg), on skeletal disease progression in MPS VII using the naturally occurring canine model. "

01/01/2018 - "A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease."

01/01/2020 - "Vestronidase alfa (recombinant human beta-glucuronidase) is an enzyme replacement therapy (ERT) for Mucopolysaccharidosis (MPS) VII, a highly heterogeneous, ultra-rare disease. "

02/01/2018 - "Benznidazole for pediatric patients with Chagas disease; vestronidase alfa-vjbk (Mepsevii) for adults and children with mucopolysaccharidosis type VII; and hepatitis B vaccine (recombinant), adjuvanted (Heplisav-B), for the prevention of infection caused by all known subtypes of hepatitis B virus in adults."