Vestronidase Alfa: A Review in Mucopolysaccharidosis VII

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

(2018) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Molecular genetics and metabolism critical review of current MPS guidelines and management

(2018) Molly Stapleton et al.   MOLECULAR GENETICS AND METABOLISM

Haematopoietic stem cell transplantation for mucopolysaccharidosis type VII: A case report

(2018) Luisa Sisinni et al.   PEDIATRIC TRANSPLANTATION

Recent progress in gene therapies for mucopolysaccharidoses

(2018) Virginia Maria Ginocchio et al.   Expert Opinion on Orphan Drugs

Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials

(2018) Yulan Qi et al.   CLINICAL PHARMACOKINETICS

Clinical course of sly syndrome (mucopolysaccharidosis type VII)

(2016) Adriana M Montaño et al.   JOURNAL OF MEDICAL GENETICS

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient

(2015) Joyce E. Fox et al.   MOLECULAR GENETICS AND METABOLISM

Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial

(2015) Christian J. Hendriksz et al.   MOLECULAR GENETICS AND METABOLISM

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

(2014) Christian J. Hendriksz et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

(2013) Joseph Muenzer   MOLECULAR GENETICS AND METABOLISM

Human β-Glucuronidase: Structure, Function, and Application in Enzyme Replacement Therapy

(2013) Huma Naz et al.   REJUVENATION RESEARCH

Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome)

(2009) Shunji Tomatsu et al.   HUMAN MUTATION